Biopharmaceutical company PTC Therapeutics (Nasdaq: PTCT) has received conditional marketing authorization from the European Commission for its drug Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older.
Sarepta Therapeutics’ new headquarters in Cambridge
Sarepta has tested eteplirsen in 12 boys so far and is ramping up studies as it moves toward approval from the Food and Drug Administration, expected next year. “Eteplirsen may hold the promise to change the face of this terrible…
Why babies should be screened for life-threatening Duchenne at birth
Penny Southall has a 14-year-old son. He’s called Dan, he goes to the local secondary school and like a lot of schoolboys, he loves computers. But unlike most of his friends, he is now wheelchair bound and may not live…
who are suitable to use Translarna with Duchenne
A drug to treat a particular form of Duchenne muscular dystrophy has been given the green light by the European Medicines Agency and could be available in the UK in six months. Translarna is only relevant to patients with a…
DMD drug Translarna gets approved by EU regulators
European Union regulators say they have given approval for the first-ever drug for treating a particular form of muscular dystrophy that affects children. Translarna, also identified under the name ataluren, treats Duchenne muscular dystrophy by helping the human body create…
Tadalafil and sildenafil alleviates restored functional with DMD
Results of a proof-of-concept study indicate that drugs such as tadalafil (Cialis, Eli Lilly) and sildenafil (Viagra, Pfizer) that inhibit phosphodiesterase type 5 (PDE5) may represent a promising new treatment strategy for boys with Duchenne muscular dystrophy (DMD). Functional sympatholysis,…
Boy with muscular dystrophy gets help to hike dream
Anthony Castle of Arizona wanted to dip his toes into the Colorado River at the bottom of the Grand Canyon. So his family and a crew of volunteers carried him down there. A 14-year-old boy who can’t walk nonetheless made…
Muscular Dystrophy Life Expectancy
Muscular Dystrophy Life Expectancy Duchenne’s Muscular Dystrophy (DMD) This is the most common form of MD affecting only boys. Since, this is an X-linked genetic disorder, males are the victims of it. However, females may carry the defective gene and…
becker muscular dystrophy life expectancy
The main features of BMD are muscle weakness and muscle wasting (muscle loss). BMD is a progressive condition, with muscle weakness becoming more severe as a person grows older. Most individuals with BMD start to experience muscle weakness or cramping…
sex and disability with muscular dystrophy
Most types of muscular dystrophy and related neuromuscular conditions do not affect sexual function or desire. And while muscle weakness may make positioning and having sex more challenging, there are many aids that can help. Discussion with a partner and…