The symposium took place on 18 th March 2025, as part of the Muscular Dystrophy Association (MDA) Clinical & Scientific ...

So it's refreshing to see the resilience of the human spirit. Muscular dystrophy is untreatable as a misconception, and I think that even in those that don't have a targeted genetic therapy ...

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Health Secretary Neil Gray is facing a furious backlash after failing to respond to an urgent plea to help children with muscular dystrophy access a new life extending drug. The Sunday Mail told ...

With grace that extends far beyond the pageant stage, Miss Asia Pacific World 2013 Srishti Rana recently took to Instagram to champion a cause close to countless hearts—raising awareness for children ...

Aaron was born with muscular dystrophy and suffered a stroke in 2023, then spent much of the following year recovering in the Midwest because of Maui's limited medical resources. "It was beyond ...

Its lead drug, EDG-5506, aims to prevent muscle damage in patients with Duchenne and Becker muscular dystrophy. The Boulder, Colorado-based company also develops EDG-7500 for hypertrophic ...

DMD is one of four conditions known as dystrophinopathies. Three other diseases that fall into this group are: Becker muscular dystrophy or Becker Muscular Dystrophy (BMD), this is a mild form of DMD ...

for the treatment of myotonic dystrophy type 1 (DM1), an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease ...

Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada The Canadian regulatory submission of vamorolone ...

The second strategy is based on insights from a similar disorder, Becker muscular dystrophy (BMD), the mutations of which preserve the transcript reading frame and result in mini- or micro-dystrophin ...

Suneel Ram’s needs gradually became greater after he was diagnosed at age 3 with Duchenne muscular dystrophy, a muscle-wasting genetic disorder that afflicts one in 5,000 boys. Today ...