advocacy

Newborn Screening Recommended for Pompe Disease

The Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) today voted to add Pompe disease (acid maltase deficiency) to a list of diseases that it recommends states screen for in newborns.

Government Funding for NMD Research Largely Spared in Continuing Resolution

The news is generally favorable for many important government-funded neuromuscular disease research projects, in the wake of Congress’ recent passage of a continuing resolution funding the government through the end of September. 

Most government operations, except those funded through the full-year appropriations bills, will be maintained at essentially the same level as fiscal year (FY) 2012. (The government's fiscal year begins on Oct. 1 and ends on Sept. 30 of the following year.)

MDA and ALSA Urge FDA to Take Action on Groundbreaking ALS Hearing

The Muscular Dystrophy Association and The ALS Association (ALSA) submitted a joint comment to the U.S. Food and Drug Administration (FDA) today, commending and thanking the agency for convening its first-ever ALS public hearing on Feb.

One Simple Thing You Can Do for Rare Disease Day 2013

In the United States, a disease or disorder is defined as “rare” when it affects fewer than 200,000 Americans at any given time; in Europe, when it affects less than one in 2,000 people.

Clearly, we’re talking about very small numbers of people. But put them all together, and the numbers tell a different story. More than 6,000 rare diseases affect more than 60 million people in Europe and the U.S. alone — some 100 million people worldwide.

FDA Hearing Highlights ALS Drug Development Concerns

Update (Feb. 28, 2013): The daylong FDA ALS webinar has been archived and is now available for viewing on the FDA site.

FDA To Webcast Its Public Hearing About ALS

The U.S. Food and Drug Administration (FDA) will host a public hearing Monday, Feb. 25, 2013, from 9 a.m. to 5 p.m. Eastern time, in Silver Spring, Md., to discuss the development of drugs for amyotrophic lateral sclerosis (ALS).

Strong Community Support Essential to Passage of Critical Muscular Dystrophy Legislation

Note: The MD CARE Act is a critical part of the historic effort to find cures for muscular dystrophies. Watch for articles in the coming weeks that delve further into the remarkable progress attributable to this important legislation.

FDA To Hold ALS Public Hearing: Input Encouraged by Feb. 8

The Food and Drug Administration (FDA) will hold a public hearing on amyotrophic lateral sclerosis (ALS) on Feb. 25. It will be open to individuals and caregivers affected by ALS, ALS clinical research experts, and those with strong opinions about the needs of the ALS community.

Federal SMA Project Issues Progress Report

A small-molecule drug candidate for spinal muscular atrophy (SMA) and an effective strategy for advancing therapy development are the primary outcomes being reported by the Spinal Muscular Atrophy (SMA) Project, a program established in 2003 by the National Institute of Neurological Disorders and Stroke (NINDS) to accelerate the development of therapeutic candidates for thi

MDA Building ‘Transitional Freedom’ Partnerships

Like many high school seniors, Drew is busy choosing a college and preparing for the big transition from life at home to life on a college campus. Unlike his peers, Drew has Duchenne muscular dystrophy.

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