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.

http://mldfoundation.org/donations.php

 

Thank You!

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 … but this month we had an exceptionally busy time supporting families as well. 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.

http://mldfoundation.org/donations.php

 

Thank You!

Always Thankful !!!

We are thankful …

  • for you,
  • your support,
  • and all of the MLD progress this year!

Teryn and I are so thankful for the wonderful people we meet as we both live with MLD (Lindy turned 38 this year!) and support families with MLD. We’ve met dozens of new families this year, many in person, as we answer questions and provide guidance on therapies and clinical care.  We’ve followed the journey with other families who lost their loved ones this year –while sad, it’s a great time of sharing and support. And, we are working with an even better and bigger team of researchers and biotech/pharma partners to get therapies to market!


We’ll provide more details on 2018’s highlights in our December update, but here are a few key items:

  • We are working with yet another drug company on a MLD therapy … and one therapy might see a 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.

If your inbox is anything like mine, it’s full of requests for support.  Nevertheless, we want to gently remind you that we need your support too! .. and we want to make it easier than ever, too.

Amazon Smile donations are FREE to YOU and are now easier than ever!  In the past, we have asked you to bookmark and start your shopping at  https://Smile.Amazon.com every time you shop at Amazon.  When you start there Amazon donates 0.5% of the value of your order to MLD Foundation at no cost to you.  While this still works, it turns out that most folks forget to start at the Smile page.  Now we have a couple of browser extensions you can load and they will automatically make sure we get credit for your order every time.  Simply load them once and shop!

Of course, a regular donation is always appreciated.

YOUR generous help makes our work possible. All of your donation goes to advance our We C.A.R.E.™ mission … no salaries are paid at MLD Foundation.

Thank You!


Safari Extension Installation Instructions.

Please note that we are working to make this process automated and IOS compatible. Right now it works only on laptops and desktops.

  1. Go to Safari->Preferences->Advanced
  2. Check “Show Develop menu in menu bar”
  3. Go to Safari->Show Extension Builder
  4. Click on the “+” on bottom left and choose “Add Extension…”
  5. Choose the KeepOnSmiling.safariextension folder inside the folder you just downloaded using the provided link (inside your Downloads folder).
  6. Choose “Install”

For more information visit MLDfoundation.org or email us at info@MLDfoundation.org