Month: October 2012

Categories
Action Alert Policy

FDA … 20 Diseases Being Chosen for PDUFA-V Meetings

Last Thursday, October 25th, I attended the FDA’s Patient-Focused Drug Development public meeting in Washington, DC. This meeting was required by the recently passed FDASIA/PDUFA-V legislation (see Abbreviation Decoder at end of post) and requires the FDA to hold 20 disease-specific meetings over the next 5 years to discuss topics such as:

“the impact of the disease on patients, the spectrum of severity for those who have the disease, the measures of benefit that matter most to patients, and the adequacy of the existing treatment options for patients.”

There were about 150 people in attendance and just over two dozen of us gave public testimony. The FDA is trying to get a better perspective on the needs, desires, and concerns of the patients by holding these 20 meetings.  Since we are directly impacted by the diseases we are best postured to tell them what’s most important to us.

Get involved … your letter to the FDA (see sample below) is needed by the end of the day November 1st! 

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Categories
Awareness Education General Research

List of Rare Diseases

This list was prepared using data supplied by the NIH’s Office of Rare Disease Research in late 2011. Please note that your medical providers and insurance payors probably have their own lists of diseases they think are rare – this list is for quick reference only. 

Quick Jump: A B C D E F G H I J K L M N O P Q R S T U V W X Y Z #

Rare Diseases and Disorders Starting With “A”

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  • Aagenaes syndrome
  • Aarskog syndrome
  • Aase Smith syndrome
  • ABCD syndrome
  • Abderhalden Kaufmann Lignac syndrome
  • Abdominal aortic aneurysm
  • Abdominal chemodectomas with cutaneous angiolipomas
  • Abdominal cystic lymphangioma
Continue reading “List of Rare Diseases”
Categories
General

Our new blog …

Welcome to the MLD Foundation’s new blog!  We’re excited to share about topics of interest to the MLD, lysosomal disease, leukodystrophy, and frankly, the entire rare disease community.

Many of you in the general public and the MLD Family know us from the family support we provide to families affected by MLD … MLD Family Conferences™, MLD Family Gatherings™, the MLD Family Discussion List™, the MLD Foundation web site, etc., but that is only a part of what we do!

We spend a lot of “behind the scenes” time working with researchers (both academic and at industry) and an increasing amount of effort affecting policy.  We are active in Newborn Screening, FDA policy (PDUFA, FDADSIA, compassionate access, Patient representative Program, etc.), NIH projects (RDCRN, CPAG, NCATS, GRDR, ORDR, etc.), educating and impacting Capital Hill (we can’t “lobby” but our voices are heard loud and clear on funding, sequester, and impacting new legislation), a slew of rare disease activities (registries, biobanks, World Rare Disease Day) and the list goes on.

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