Duchenne Muscular Dystrophy (DMD)

DMD — Catabasis Pharmaceuticals

The Muscular Dystrophy Association has awarded $120,000 to Cambridge, Mass.-based Catabasis Pharmaceuticals. The award is part of a strategic partnership under which the pharmaceutical company will test two compounds — CAT-1004 and CAT-1040 — in the mdx research mouse model of Duchenne muscular dystrophy (DMD).

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.

DMD: Drisapersen Receives ‘Breakthrough Therapy’ Designation

Multinational pharmaceutical company GlaxoSmithKline (GSK) has announced that its experimental Duchenne muscular dystrophy (DMD) drug drisapersen has received breakthrough therapy designation from the U.S. Food and Drug Administration.

DMD: Eteplirsen Shows Continued Benefit at 84 Weeks

Eteplirsen, an experimental exon-skipping drug designed to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show sustained benefit on walking distance through week 84 of a phase 2b, 12-person study.

DMD Symptoms Show Up Early and Can Be Measured, Study Finds

Careful assessments of motor, cognitive and language function in very young children with Duchenne muscular dystrophy (DMD) show these children lag behind their age-matched peers well before obvious symptoms of the disease appear.

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: 'Permanent' Gene Repair Strategy Looks Good in Lab

Permanent repair of a faulty gene has long been a goal of researchers working to develop gene-based therapies. But many current gene modification strategies that have entered clinical trials have been based on temporary forms of gene correction — treatments that will need to be given frequently throughout a person's life.

New Muscular Dystrophy Drugs Offer Hope

Moving Toward 'Next-Generation' Gene Therapy

Planning for the next generation of gene and stem cell therapies for muscular dystrophies — even as the first generation is still under development — was the theme of a joint symposium sponsored by MDA and the Association Française Contre les Myopathies (French Association Against Myopathies, or AFM) at the 16th annual meeting of the American Society of Gene & Cell Therapy (ASGCT).

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