Roche: spinal muscular atrophy drug meets trial goal
(CercleFinance.com) - A pivotal trial of Roche's experimental drug risdiplam found that it significantly and meaningfully improved motor milestone in babies with type 1 spinal muscular atrophy, it said.
The study's primary outcome measure was the proportion of infants aged 1-7 months sitting without support for at least five seconds after 12 months of treatment.
To date, over 400 patients have been treated with risdiplam across clinical trials, with no safety findings leading to study withdrawal, Roche said.
Spinal muscular atrophy is a progressive neuromuscular disease that causes muscle atrophy. It is one of the most common rare diseases, affecting approximately one in 11,000 babies.
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The study's primary outcome measure was the proportion of infants aged 1-7 months sitting without support for at least five seconds after 12 months of treatment.
To date, over 400 patients have been treated with risdiplam across clinical trials, with no safety findings leading to study withdrawal, Roche said.
Spinal muscular atrophy is a progressive neuromuscular disease that causes muscle atrophy. It is one of the most common rare diseases, affecting approximately one in 11,000 babies.
Copyright (c) 2020 CercleFinance.com. All rights reserved.