MLD Moms are the Best!

Happy Mothers Day

We’ve met hundreds of you face-to-face, talked to many of you on the phone for hours, and have met many many more through the Internet.

One thing that stands out is the moms. Yes, there are strong dads, grandparents, extended family, and friends … but it’s the moms that stand out.

mom with baby in grassy field

We’ve met moms of all circumstances … some of your loved ones are living while others are MLD angels, some where MLD is now or in the recent past and some where it has been decades, differences in where you live and how you live, some started with lots of medical knowledge while most started from scratch, some with access to great care and support while many have one or neither, some with financial resources of their own or available to them while most have moderate to no financial resources, some who were blessed to access life-changing therapies for their loved ones and others that couldn’t … and the list of differences goes on and on.

elephant with calf

But what is most significant to us is what you share in common … love, compassion for one another, strength when you think you don’t have any left, hope in your heart when every bone in your body says there is no hope, the tiger mom instincts you show when problem-solving, the ability to do more than you ever thought on less sleep than you ever thought you’d need, you are jugglers of care and compassion for the rest of your children and family as well as your MLD loved one(s), … and show sparkles in your eyes even when the sparkle is through tears.

… and show sparkles in your eyes even when the sparkle is through tears.

We’ve been with you through the search for a diagnosis, the panic of diagnostic confirmation, the search for appropriate and accessible therapies, optimizing quality of life through clinical care, garnering social services and school-based supports, transitions for some of your loved ones to young adulthood, transitions as spouses were consumed by MLD, sharing as your loved ones lose skills and abilities, and for many, walking alongside you those last days for your loved one – and the days that follow for you as you adjust to a different life with a huge hole in it.

mom with child ocean sunset

All the way along this journey, moms, you are an encouragement to others and to us. You show kindness, strength, and most importantly, love … to your family, those around you, and to us. For that we give thanks.

Butterfly hugs,

Dean & Teryn
… and Lindy too!

MLD Foundation butterfly logo with tail
Advocacy Awareness Clinical Care General Research Therapies

MLD Gene Therapy Approved in Europe

Gene therapy for early-onset MLD patients has been approved in all 27 EU member states as well as the UK, Iceland, Liechtenstein, and Norway for early-onset (late infantile and early-juvenile) patients.

Congratulations to the San Raffaele, TIGET, and Orchard Therapeutics teams, the clinical trial families, and the entire MLD community on this significant milestone! MLD Foundation first started working with the San Raffaele/TIGET researchers in 2005 and has been fully engaged and actively supporting the project throughout its pre-clinical phase and subsequently nearly a decade of human trials. The results in the target patient population have been outstanding. We are excited to continue on the journey toward approval & access across the EU … and throughout the world.

Dean Suhr, President & Co-Founder, MLD Foundation

Following the receipt of a European Medicines Agency (EMA) positive opinion last November recommending full marketing authorization for Libmeldy™, the gene therapy becomes the first gene therapy for eligible patients with early-onset MLD, and MLD’s first commercial therapy.


Presymptomatic late-infantile and early-juvenile forms of MLD are included in the approval. The approval also includes early-juveniles with early clinical manifestations (symptoms) who are still able to walk independently and have not started to decline cognitively.


Orchard has a clinical trial underway studying Libmeldy in the late juvenile form of MLD, however, it should be noted the late juvenile and adult forms are not covered by this commercialization approval.

Patients outside the EU and with later-onset forms of MLD are able to request Orchard for access to Libmeldy via Orchard’s Pre-Approval Access/Compassionate Use programs. Orchard reviews each request on a case by case basis. MLD Foundation will help you advocate for access for your loved loved ones.


Access and reimbursement … EMA and EC approval is a huge milestone, but it is not the finish line. MLD Foundation will continue to work directly with Orchard, EU partners, and country officials as each EU member country undertakes its own Libmeldy price and reimbursement review to make the therapy formally made available to its citizens.

FDA (USA) … MLD Foundation has been in communication with the FDA and is in full support of Orchard’s IND activities that we expect will lead to a BLA request for review and eventual approval in the US.

Newborn screening … MLD Foundation is leading an MLD newborn screening development, validation, and implementation effort to identify patients pre-symptomatically. We need your participation to make this effort successful. Click here to learn more and join us!

Subscribe to this blog to be kept updated on gene therapy and other therapeutic advances, and ongoing efforts to improve other aspects of MLD quality of life.

Orchard Therapeutics Press Release

* Libmeldy is the EU commercial name for the gene therapy and is a trademark of Orchard Therapeutics.
* The image on this page is a screen capture of Orchard Therapeutic’s homepage featuring one of the first MLD gene therapy patients.

Action Alert Compassion General

Giving Tuesday – It’s Our Day

Did you know that today, Tuesday, December 1st, is Giving Tuesday?

After a month of “Black Friday” and yesterday’s “Cyber Monday” frenzy, it’s a day to focus on our loved ones!

We aren’t asking for your donations …

MLD Foundation knows that MLD families have their hands full with their loved ones so for the past two decades we’ve made it our practice to not directly ask you to be our primary source of financial support.

But with that said, we are so thankful for the many families over the years who have organized events and fundraisers … and we are so humbled to be remembered when loved ones get their angel wings.

We are asking you to please share with your friends, extended family, …

Today we’re asking you to share with your extended family, friends, neighbors, co-workers, and social networks how important and impactful MLD Foundation has been to your family. We need your help to ask for their support!

How we use donations …

We are frugal with all donations … they go directly to expenses surrounding our core We C.A.R.E.™ mission …

We C.A.R.E.™ … Compassion for families, increasing Awareness, influencing & funding Research, and promoting Education for metachromatic leukodystrophy, a very rare terminal genetic neuro-metabolic disease where over half the cases affect infants.

Since our founding, we have not had any paid staff, all of our work has been a labor of love … no donations have gone to salaries.

2021 will be busy and productive

As we highlighted in last week’s Thanksgiving blog, 2020 has been a very productive year, and 2021 is looking to be the same.

We have a major multi-year project underway making newborn screening a reality for all families worldwide so children have the broadest and optimal choice of therapies while their loved ones are pre-symptomatic and therapies are most effective.

But that’s not all – we are working to assure access and reimbursement for the gene therapy emerging in the EU, we’re supporting the submission and review of gene therapy by the FDA in the USA, increasing physician and family awareness, improving clinician education, developing an MLD Standard of Care, and supporting a number of new therapies in development and under clinical trial at nearly a half dozen biopharma companies, and so much more.

Please share … a few moments of your time will allow us to to keep serving you!

Compassion General

We’re thankful – even in 2020!

We heard earlier this week that nearly 4 in 5 people are already looking forward to putting 2020 behind us. The pandemic (family safety, work, school, loss of loved ones, individual and community response, social isolation, etc.), US elections, income, health, … it is affected every one of us, and it’s overwhelming.

But we are still thankful…

… not for MLD, not for loss, but for all of you – our friends, family, the MLD Family™, the greater MLD community, the MLD researchers and clinicians, regulators, policymakers, and the rare disease ecosystem who supports us.

The MLD Family™ …

… is supporting each other. There is advice, compassion, and the sharing of excess or needed supplies. We can’t meet in person, but we’re connecting with regular men’s, women’s, and now couple’s calls and so much more as we adapt to new ways of connecting.

MLD Research is progressing …

Orchard Therapeutics has achieved EMA/CHMP recommendation for approval of gene therapy in Europe. They have also filed an IND with the US FDA, a key stepping stone formalizing communications with the FDA as they move one step closer to US review and approval.

Takeda continues its ERT clinical trial. Homology Medicine continues to make pre-clinical progress with their gene therapy. And we are working with several other biopharma companies, too!

MLD Newborn Screening …

We have to diagnose children earlier in order for them to have good viable life-saving therapy options!

The MLD Newborn Screening program is making great progress. New York is right on the cusp of launching an MLD pilot study into parts of their state. The MD NBS Expert Advisory Group is meeting monthly. Many of you are participating with the Working Focus Groups – you can still join us for our December meetings!

MLD Cinical Care Continues to Improve …

We are blessed to have many wonderful and knowledgable MLD clinical researchers … we still need more, but those who do support our community are open, supportive, and often go the extra mile as they care for our loved ones in person or remotely. We worry about their health as they are called into care situations where additional COVID resources are necessary – let’s all keep doing our part to keep these COVID care needs minimized and our clinicians safe!

In 2020 we started to lay the foundation for creating an MLD Standard of Care so we can all more easily get approval and reimbursement for better, more consistent, and more appropriate MLD clinical care.

Let’s keep connected …

Subscribe to this blog to be kept updated on therapeutic advances and ongoing efforts to improve other aspects of MLD quality of life.


Gene Therapy Recommended for Full EU EC Marketing Authorization Review

Gene therapy for early-onset MLD patients passed a huge milestone yesterday when the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy, by the European Commission (EC), which has the authority to grant marketing authorization for Libmeldy* in the European Union (EU). The EC is anticipated to make a final decision by the end of 2020. If approved, Libmeldy™ would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.


Presymptomatic late-infantile and early-juvenile forms of MLD are included in the recommendation. The recommendation also includes early-juveniles with early clinical manifestations (symptoms) who are still able to walk independently and have not started to decline cognitively.


After the EC marketing authorization is approved, each EU member country will have to do its own Libmeldy price and reimbursement review before the therapy is available to its citizens.

MLD Foundation is leading an MLD newborn screening development, validation, and implementation effort to identify patients pre-symptomatically. We need family participation to make this effort successful. Click here to learn more and join us!

Subscribe to this blog to be kept updated on gene therapy and other therapeutic advances, and ongoing efforts to improve other aspects of MLD quality of life.

Orchard Therapeutics Press Release

* Libmeldy is the EU commercial name for the gene therapy and is a trademark of Orchard Therapeutics.

Awareness Clinical Care Compassion Education General Newborn Screening Policy

We have 20/20 Vision for MLD

2020 will bring new clinical and therapeutic care to the MLD community.

When we started serving MLD Families in 1999 – two decades ago – we had no idea how challenging and time-consuming it would be to improve clinical care and disease altering therapies.

Along with your support, we’ve done a lot of heavy and consistent lifting these last 20 years.

If you read nothing else of this blog, please know that we gratefully THANK YOU and wish you and the MLD community a Happy New Year!!

Our Time is Now!

We look forward to reaping the benefits of these past two decades and our ongoing work during 2020 and the first couple of years in this new decade. During 2020, after many years of research, pilot studies, and clinical trials, families will start to benefit directly as this work becomes available to families to improve the quality of life for those with MLD.

NEWBORN SCREENING … Newborn screening is critical to identifying MLD at the earliest possible time – birth. These infants will have the greatest benefit from current and new disease-altering therapies.

We’ll see the launch of newborn screening in the first US public health system in parts of the state of New York as a precursor to a federal RUSP application, screening in more US states, and adding newborn screening to various international health systems too!

Teryn Suhr of MLD Foundation took the lead to develop the screening technique that is being used today. We’ve led in instituting powerful state NBS policy in California and Florida. We’ve hosted a Newborn Screening Summit for MLD experts and will host several more this year. We are continuing and expanding work as we are coordinate and manage a federal RUSP nomination as well as state-by-state & country-by-country MLD newborn screening implementation.

Early diagnosis through newborn screening and an expanding choice of therapies means each family can choose what is best for their child.

GENE THERAPY … gene therapy clinical trials have shown extraordinary results with pre-symptomatic children. Orchard Therapeutics filed with the EMA this past fall and should have their response – hopefully, an approval – around mid-year. We hope the US FDA review will start in early 2021 – maybe sooner!

Homology Medicines is seeing excellent pre-clinical research for its novel approach to gene therapy.

MLD Foundation is and has been working with at least four other pharmaceutical companies and research spinouts but cannot share more about their pre-clinical work (yet) due to NDA requirements. We speak regularly with startup companies and venture capital companies about the opportunities and challenges surrounding MLD.

And there is more …

STEM CELL TRANSPLANT … we are seeing ongoing improvements in MLD stem cell transplant, MLD’s only widely available therapy. In addition to the studies underway at various hospitals and academic institutions to reduce mortality, reduce chemotherapy, and improve outcomes for current MLD transplants, Magenta Therapeutics has a clinical trial studying cell-expansion and is working in the lab to eliminate chemotherapy as part of the transplant process.

BETTER DISEASE UNDERSTANDING … There are over 300 mutations known to cause MLD – that’s 90,000 potential mutation combinations. We only know the progression of a handful of those mutation pairs with decent certainty. MLD Foundation is actively engaged in a genotype-phenotype study to better help families make optimal informed quality of life clinical and therapeutic decisions.

We’re also active in two academic Natural History studies and operate an MLD Patient Powered Registry where MLD families are sharing their MLD journies so we can improve not only the medical care, but also improve the social, emotional, financial, public services, and creative family-driven/inspired quality of life strategies and decisions.

We have been meeting with the FDA since 2007 to expand their understanding of MLD and the therapeutical needs of the community. As we get closer to a regulatory filing we will both focus and expand the communications with the agency.

But not everything about living with MLD is medical

COMPASSION AND SUPPORT FOR FAMILIES … We do our best to address the needs of MLD families in hands-on practical ways … some recent examples of direct engagement: we’ve helped several families get wheelchair vans for their loved ones, We’ve re-purposed medical equipment, provided logistical and financial support to help a family get same-day passports to get to Italy for gene therapy evaluation, helped overturn MEDICAID out of state therapy rejections, visited Italy to help a family enroll in the gene therapy, helped families make numerous therapy and non-therapy decisions, appeared at a deposition to help a family get in-state MEDICAID support for their family.

But perhaps the most impactful work we do is when we meet with families in person, at conferences, and over the phone. As we all know, an MLD diagnosis is just the start of a journey filled with decisions impacting all facets of life all while grieving the impact of MLD on a loved one and our parental/spousal expectations for life.

We spend hours on the phone with many of the newly diagnosed families helping them to learn about the disease, what to expect, clinical and therapeutic decisions, life decisions, and just being there with them as they digest an overwhelming amount of information and make decisions no parent or spouse should ever have to make.

And of course, we have host an annual MLD Family Conference™. This is where you can meet with dozens of other MLD families face to face with extended time for conversation. There are always MLD clinical and research experts to engage as well. In 2019 we launched the MLD Newborn Screening Academy. In 2020 we will continue that education and empowerment. MLD cannot reach the finish line without your direct efforts in your home state.

How Do We Do It?

We do all of this with your support … financial, time, energies, introductions to your connections and resources, “tips” about new ideas, approaches, and activities, and with the support of your thoughts and prayers.

Our philosophy has always been to try to meet researchers, companies, regulators, policy-makers, investors, other advocacy organizations, and most importantly, families, in person whenever possible. We visit them in their offices, labs, hospital rooms, and homes. We have found that we can have more intimate, detailed and informative conversations in person than via email or phone. We make it a point to share whatever information we can to connect the knowledge, needs, and experiences of one person with another … and sometimes that includes finances too! We’ve seen dramatic results and huge leverage with this strategy.

As you can see below, it’s a lot of nights on the road on your behalf. Life with MLD never stops so there are no days off … we are always honored to be working for you as our mobile phones and laptops travel with us.

The total is since 2008, when we started using TripIt to manage our travel.

In addition to face-to-face meetings, we spend extensive time in Washington DC with the FDA, NIH, other advocacy groups, and policy-makers. There are a half dozen or so key conferences and events each year. However, we don’t just attend those conferences, we also actively participate by presenting, booth displays to educate, posters to educate, and participating in panels to inspire.

MLD PATIENT POWERED REGISTRY … This is how our experiences are being gathered to impact researchers, clinicians regulators like the FDA, and policymakers. Unlike most natural history studies that only ask a small number of families a limited and focused set of disease-specific medical questions, the MLD PPR covers all aspects of life with MLD … from diagnosis to extended family, finances, clinical care, social services, and much much more.

As we gather more data we gain breadth, understanding, and perspective. All of this allows us to have a greater impact as we use the data. Researchers can request access to the de-identified data so they can efficiently advance their work.

We want all families to participate … no matter how recent or long ago your MLD experience is, no matter if your loved one is living or has passed, no matter what your home country is – everyone has MLD journey experiences to share and for us to learn from.

How Can You Help?

Reach back to us and let us know you are interested. Every one of you has skills that enhance or expand what we can do, knows people we don’t, and lives in a different part of the country or the globe.

Got a minute? 2 minutes? 60 minutes? We will be blessed by just a minute or two whenever you can spare them.

We recognize that your emotional and physical needs will ebb and flow as you navigate the MLD journey. We know that too, so we will always try to make sure you are supported and not overwhelmed.

Do you love social media? We have a special need for someone or multiple people to help us with our social media.

Dean Suhr
MLD Foundation
21345 Miles Drive
West Linn, OR 97068-2878 USA

1+503-656-4808 or 1-800-617-8387 Serving the MLD Community since 2001We C.A.R.E.™ … We are the largest charity worldwide focused on MLD family Compassion, Awareness, Research and Education.


Super Bowl Alternative – MLD Registry

Are you bored waiting for the SuperBowl pre-game? Or twiddling your thumbs while waiting for the next commercial to air?

If so, please take a few minutes and start to enter your data into the new MLD Patient-Powered Registry. We want to understand MLD better, accurately characterize the MLD patient, family, and caregiver journey, justify newborn screening, improve clinical care and of course, to improve research and approval of new therapies. We can’t do this without you!

YOU WILL GET SOMETHING BACK FOR EVERY QUESTION YOU ANSWER … the survey is designed to report back the results of everyone else’s answers as you complete each question so you will know if you are typical or a bit different than other MLD families. Of course, the first few of you filling in the survey won’t have a lot of other family’s data to look at but after the first 10 or 20 of you participate it will become a rich dataset.

WE NEED EVERYONE TO PARTICIPATE … Please note that we want to know about your MLD loved one(s), including MLD angels that live with us in our hearts. By sharing your experiences and data your loved ones will be part of changing the world for those that haven’t started the MLD journey.

YOU CAN PARTICIPATE if your loved one HAS ANOTHER LEUKODYSTROPHY or LYSOSOMAL DISEASE … We won’t ask you to fill out the MLD specific questions, but there is plenty to learn from the broader community as well.

We’ve been working a long time to get this survey launched … there are modules for you to complete at your own pace on demographics, diagnostics, therapies, cannabis, awareness, newborns screening and

You own your survey data and will always have 100% control over it. MLD Foundation will manage your data and creates the survey modules, Genetic Alliance developed and is hosting the survey platform, and Private Access will make sure that it is always secure.

Over the next few days we will tell you more about the survey and how we are using the data you provide.

At the Super Bowl we know there will be a winner and a loser … but rest assured that we all will win if you participate in the MLD Patient-Powered Registry!

Thank you!


5 Biotechs Actively Developing MLD Therapies

2019 is going to be a very interesting and productive year for MLD therapy development. The trials for both gene therapy and enzyme replacement therapy will be expanded and for the first time are headed towards sites in the United States. A third biotech has publically announced MLD is an active development candidate.

We are getting ever closer to a NDA* filing for review (and hopefully approval) with the FDA and other global regulatory agencies.

Last December we met and strategized with four pharma/biotech companies who are actively working on MLD therapies. For now, we cannot name one of those companies or talk about what they are working on but we will tell you about the others plus a fifth parter we are also working with. There is a sixth company under wraps – but that’s for another time.

MLD Foundation spends considerable effort working with academic, institutional, and corporate researchers around the globe helping to advance MLD basic research, influence clinical trial design and strategies, and expediting clinical trial development and recruiting. We are equally active and aggressive in diagnostics, including newborn screening, and policy related to regulatory approval, access, and reimbursement (including pricing). Our view certainly starts in the USA, our home country, however, we have a global footprint ad active connections from Australia, through southeast Asia, and across Europe.

TAKEDA (SHIRE) – Enzyme Replacement Therapy (ERT)
The acquisition of Shire by Takeda was completed yesterday (Jan. 8th). Part of Takeda’s interest in Shire was its extensive rare disease portfolio, including its lysosomal disease portfolio where is where the MLD ERT program resides. The company name is now Takeda, but the team working on MLD remains intact and is expanding slightly. Shire will open the next phase of their ERT trial in April with global recruiting of 35 late infantile patients including 10 sites here in the USA.

Orchard TherapeuticsGene Therapy
Orchard Therapeutics gene therapy program has an active trial underway (also for late infantiles) working to validate the freezing of the genetically modified material for transport to and from the Italian processing facility. This process has been validated previously by another company so it’s likely it will work. Once that is complete we expect Orchard to open global sites for future phases of their clinical trials, including sites in the USA.

Homology MedicinesGene Therapy
Homology Medicines has been working on MLD for quite some time without much public fanfare. Their gene therapy therapeutic approach is being validated in PKU. MLD was just given the go-ahead by their Board as their lead central nervous system product and will head into IND-enabling* studies. Homology has just moved into a new facility and will be doing their gene editing on site in their new manufacturing facility.

Both Orchard and Homology had initial public offerings in the US last year that are enabling their programs to progress quickly.

And a fifth biotech partner, Armagen, continues their clinical trial on another lysosomal disease alongside pre-clinical basic science work on MLD.

We will be sharing more on each of these programs in the coming days and weeks including how they might affect each of your loved ones and how you can help them move forward.

* NDA – New Drug Application is the formal application for FDA review and approval which leads to widespread availability of a new therapy or drug.
* IND – Investigative New Drug is the terminology used by the FDA as they consider an application for clinical trials in humans.


Christmas Message – to the MLD Family

It’s Christmas all over the world  … and is usually my tradition, I share some personal thoughts with all of you, my MLD Family.

As many of you know, we lost our Darcee to MLD in 1995 and while Lindy is still with us, she is losing the MLD battle bit by bit.  Tonight Teryn, Lindy and I shared a full turkey dinner … a quiet and special time.  It took a long time for Lindy to chew and swallow.  As her eating abilities decline, we don’t know, but this might be the last time she will eat a formal meal like this.  Throughout the meal she was vocalizing to us … and a couple of times it seemed she was trying so hard to get 2 or 3 words out – but we could not understand them.  We don’t know what she was trying to say, but when we snuggle with her and speak to her she always smiles so we have an alternate means of communication!
So that left Teryn and I to do the talking. We chatted for bit and then circled around to a MLD family that we know who has one child in transplant, and a second who is headed to Milan today (Christmas day) for gene therapy. We have become very tightly engaged with this family having walked the journey after being able to meet them just 48 hours after thier first child was diagnosed.

Earlier tonight at Christmas eve church service we crossed paths with a friend we had not seen for several years.  She lost one of her children to SIDS many years ago … in December.  Which reminded us of another MLD family who is very close to us who lost their daughter to MLD in December, and of course our Darcee’s angel date in December 16th.

Christmas is a time of great joy for many children and families … but it is often mixed with the sadness of a loss of a loved one – either as an angel or perhaps just the continued progression of MLD.  For those of us who are Christians there is often conflict with the expectations of joy this time of year.

Life is complicated.  And with MLD, like so many other rare diseases, the path is uncertain, and full of both expected and unexpected surprises.  Some of those surprises knock us off our feet and take our breath away replacing it with despair and sadness … and others – often simple little things – bring smiles, happiness, contentment, and joy.
So wherever you are on the journey and whatever you are feeling … I can only share what works for me … its the joy of the greater MLD Family that loves and cares for each other.  We have personally met many of you, talked with others over the phone, shared personal email conversations. I am honored to know you and your MLD loved ones.  I am honored to be able to represent you as we do the work of MLD Foundation on behalf of each of you.

Please give a hug, hold a hand, speak kindly, and/or share a smile with all of those around you this holiday season.  Find a moment (or more) of joy – I know it’s in those around you.  And especially if you are struggling, reach out.  We are all here for you!
For those of you that lost a loved one this year, or anytime, and struggle with Christmas and the holidays, I dedicate the following song to you.  May its message of love and peace give you comfort …

Merry Christmas,
Dean (and Teryn & Lindy too!)         —

Listen to the song here:

The story behind this Song:
“So last year, was a really tough year for us.  We lost our daughter, Annie.  People talk about grief, pain, fear, sorrow.  All of these words, can’t really describe how you feel when you really go through the loss of a loved one.  Even though we held onto hope, that we would somehow see her again, it was just a brutal time that we were going through, right at Christmas.  It felt pretty hard to celebrate.  
And then I found this song.  It was just a video on Facebook by this Christian artist and he was singing about the very struggle I was having.  As my wife and I listened to the words of this song, it expressed exactly what we were feeling. It also gave us what we needed at the time.  That was comfort.  So many people struggle with loss at this time of year, not just me.  If you are missing someone this Christmas, I want to dedicate this song to you.  I also want to dedicate it to Annie, my sweet Annie, who I miss very much.”-Jon Schmidt
Lyrics:I’m not going to lie.Christmas really hurts this timeCause you’re not here to celebrate with me.Tears fill my eyes.And memories flood my mind,As I place your ornament upon our tree.Although this year I have a broken heart,It gives me hope and joy as I remember where you are.  
Chorus:You’re with the Son of God.You’re with the Prince of Peace.You’re with the one we’re celebratingAnd that thought amazes me.Sometimes I still break down,Grieving that we’re apart,But the sweetest gift is knowing where you are.You’re with the Son of God.
Got your picture in a frame And a stocking with your name.Oh God knows it’s been hard letting go. And I can’t bring you back,But I’ll see you again.And oh, that thought is healing to my soul.I’ll miss making angels with you in the snow.I guess instead you will be singing with them All around God’s throne.
And I know the Christmas seasonWas your favorite time of year.You loved to help us decorate our tree.But now that you’re with Jesus,I can’t imagine how you feelCause He’s the one who bled and diedUpon the tree for you and me.
Credits: “The Sweetest Gift” written by Craig AvenProduced by Jon Schmidt and Chuck E. Myers “sea”Arranged by Jon Schmidt, Chuck Myers, Al van der Beek & Steven Sharp NelsonCraig Aven: VocalsJon Schmidt: PianoSteven Sharp Nelson: CelloPiano and vocals recorded by Jake Bowen at Big Idea StudiosCello recorded by Al van der Beek at TPG StudiosMastered by Al van der Beek
Video Produced and Filmed by Paul Anderson & Shaye ScottVideo Edited by Shaye Scott & Paul Anderson

Dean Suhr
MLD Foundation
21345 Miles Drive
West Linn, OR 97068-2878 USA

1+503-656-4808 or 1-800-617-8387 Serving the MLD Community since 2001We C.A.R.E.™ … We are the largest charity worldwide focused on MLD family Compassion, Awareness, Research and Education.

Advocacy General Newborn Screening Research

Your Giving Keeps Us Going – and directly helps families! #GivingTuesday

#GivingTuesday is Here!

Here in the US we celebrated Thanksgiving this past weekend.  It’s a time of family, thanks, and giving.  Most of us “gave” at the mall on Black Friday or online on Cyber Monday … but now it’s #GivingTuesday, an annual day where the world comes together to give.

November is MLD Foundation’s second busiest travel and meeting time … so much so that we have yet to decompress and summarize all that we have done. Your generous gifts are what enables us to work on your behalf!

We’ve Been Busy!

We’ll send out some summary postings over the next few weeks, but in the meantime, here are a few highlights of our last 60 days …

  • We are working with yet another drug company on a MLD therapy … and one therapy might see an FDA registration next year!
  • MLD gene therapy has a new owner, Orchard Therapeutics … and they are moving much more quickly than the prior owner.
  • The MLD newborn screening pilot study is making great progress and we are preparing to launch in another state in a few months.
  • Met with a pharma partner to help advance an ongoing genotype-phenotype study they have underway,  This will be important to try to determine what form of MLD progression is expected if a baby is diagnosed at birth … and potentially for those diagnosed with early symptoms too.
  • Attended the annual Rare Diseases and Orphan Products Breakthrough Summit sponsored by NORD.
  • Met with industry, BIO, and numerous advocacy organizations to drive collaboration on rare disease policy – especially for ACA and Orphan Drug Tax policy issues.
  • Met with another Pharma company who detailed to us some success and roadblocks for their therapy development.

And Most Importantly …

  • Continued our Compassion support for families … recently on the phone and in person in California, Maryland, Colorado, and even Perth Australia, and around the globe via email and social media. We’ve recently helped families get confirming diagnostics, make therapy decisions (including gene therapy, stem cell transplant, and no therapy), connect with clinical resources, obtain wheelchair vans and other equipment, and have been a source of direct financial support when unexpected expenses have arisen.

Please remember to give on #GivingTuesday to support MLD Foundation! There are no salaries paid here – all donations go directly to Compassion support for families and to advance MLD Research and Awareness.


Thank You!