Becker Muscular Dystrophy (BMD)

2013 BMD Conference Covered Research, Medical Care and Daily Life

The 2013 MDA-supported Becker Muscular Dystrophy Conference, held Aug. 24 in Baltimore and co-hosted by MDA and Johns Hopkins Medicine, was "high-energy" with "phenomenal" presenters, says Annie Kennedy, MDA's senior vice president of advocacy, who attended and helped organize the meeting.

Some 130 people attended in person and many more, including some from outside the United States, attended online.

DMD: Summit Granted Patents for Utrophin Booster

Summit PLC, a biotechnology company based in Abingdon, United Kingdom, has announced it now has two U.S. patents for its experimental drug SMT C1100 for Duchenne muscular dystrophy (DMD).

The patents will provide commercial protection to the company as it continues the further development of this drug. However, the drug will not become available to patients until it receives approval from regulatory agencies.

Becker MD Conference Can Be Attended Online Aug. 24

MDA and John Hopkins Medicine will present the 2013 national Becker Muscular Dystrophy Conference Saturday, August 24, in Baltimore. Free to all participants with BMD and their family and friends, the conference can be attended in person or online.

Nationwide Children’s Podcast Explores Therapy Development for DMD

In a July 2013 podcast from Nationwide Children’s Hospital in Columbus, Ohio, longtime MDA grantee Jeffrey Chamberlain discusses recent advances in the development of gene therapy (gene transfer) and stem cell therapy (transplantation) for Duchenne muscular dystrophy (DMD), the results of which may apply to other types of muscular dy

DMD/BMD — Lynn Megeney, Ph.D.

Lynn Megeney, senior scientist at the Sprott Centre for Stem Cell Research, Ottawa Hospital Research Institute in Ottawa, Ontario, Canada, was awarded an MDA research grant totaling $300,000 over a period of three years to study how muscle stem cells are controlled.

DMD/BMD: Questions About Ataluren’s Mechanism

It's been widely accepted that the mechanism by which the experimental drug ataluren appears to benefit walking ability in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is that it causes "read-through" of premature stop codons — genetic instructions that cause cells to stop making a protein before the process is complete.

BMD: Participants Sought for Registry Associated with Gene Transfer Trial

In connection with a gene transfer clinical trial, investigators at Nationwide Children's Hospital in Columbus, Ohio, are seeking people with Becker muscular dystrophy (BMD) for participation in a patient registry.

The investigators say the registry will provide information that will:

DMD/BMD — ARMGO Pharma

MDA has awarded $1 million to biopharmaceutical company ARMGO Pharma for development of a new strategy for treating Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).

DMD, BMD: Phase 3 Trial Opens for Stop Codon Read-Through Drug

A large-scale, multinational phase 3 trial of the experimental drug ataluren has opened its first trial site, in Cincinnati, Ohio.

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