Researchers at The Ottawa Hospital in Canada have received $150,000 from Cure SMA to develop new ways of monitoring spinal muscular atrophy (SMA) using a noninvasive blood test.
The team, led by ... Read more
Gyula Acsadi is an expert in three of the world’s most expensive rare illnesses to treat: spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and Pompe disease. Acsadi, head of ... Read more
According to a new study, a molecule known as A15/283 being developed to treat SMA , has shown significant efficacy in male mouse models of the disease.
A15/283 is a ... Read more
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