Why Does It Take So Long To Go from Mouse to Man?

John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported, where a treatment appears to cure one neuromuscular disease or another in a mouse — yet there are few treatments available today for people with any of these diseases, and only a few treatments in human clinical trials. Why does it take so long?

CoQ10, Lisinopril Trial in DMD, BMD, LGMD2C-2F, LGMD2I

Researchers at five U.S. and one Canadian center are conducting a clinical trial of the medications coenzyme Q10 and lisinopril to determine their possible beneficial effects on heart function in Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and five forms of limb-girdle muscular dystrophy (LGMD).

NIAMS Establishes MD Research Center

A Center for Research Translation of Systemic Exon Skipping in Muscular Dystrophy has been established by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the U.S. National Institutes of Health.

Caregivers: Teaching Independence

When Vance Taylor was a boy, he didn’t know any adult he could look to and say, “There’s somebody like me.”

His mother, Morena Noyes, recalls the first time she took Vance and his sister Kathy — both of whom have limb-girdle muscular dystrophy— to MDA summer camp.

“We were still in the parking lot, in our Astro van,” Noyes says. “Vance looked, and then he turned to his sister and said, ‘Kathy, there are people like you and me here!’ He was just beaming.”

Research Updates Fall 2011

PTC Remains Committed to Ataluren for Nonsense-Mutation DMD/BMD

Update (July 25, 2012): This story was updated to reflect the fact that PTC has announced its intention to begin an open-label trial of ataluren in DMD/BMD for former ataluren trial participants in Europe, Israel and Australia.

MDA Awards $13.7 Million in Research Grants

The Muscular Dystrophy Association has awarded 40 research grants totaling $13.7 million to advance the understanding of disease processes and uncover new strategies for treatments and cures of muscular dystrophy and the more than 40 other diseases in the Association's program.

The new grants were recommended by MDA's Scientific and Medical Advisory Committees and approved by MDA's Board of Directors at its July 2011 meeting.

Families and Experts Gather at 2011 BMD Conference

Community and empowerment were the themes of the third annual Becker Muscular Dystrophy (BMD) conference held at Cedars-Sinai Medical Center in Los Angeles on August 13, 2011.

Some 150 in-person attendees, and at least that many online attenders, engaged physicians, researchers, physical therapists and other BMD experts on a variety of topics, including the latest advances in research, medical management, physical therapy and clinical trials.

Hey! I'm Here Too!

It's not easy when your brother has muscular dystrophy. His illness might bring up thoughts and feelings that upset you. There may be questions you don't know the answers to. You might be shy about asking those questions and about asking for help because your brother needs so much attention.

This brochure was written especially for you. It will help you let people know, "Hey! I'm here, too!"

Last Updated: 
Fri, 04/02/1999 - 09:00

Andrew Cameron

Location

Austin, TX

Andrew’s artwork was the first piece selected for inclusion in the MDA Art Collection and exhibited at the Association’s national headquarters in Tucson, Ariz. He created the collage in a school art class in 1991. Andrew’s hobbies include digital graphics, photography and custom cars.

Full name: 
Andrew Cameron

Amusement Park

Artist: 
Andrew Cameron
Medium: 
Construction Paper Collage

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