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Bio Blast Pharma reports promising data on muscle wasting drug

Meghna A SinghaniaWritten by Meghna A Singhania Published On 2015-10-28T12:52:45+05:30  |  Updated On 28 Oct 2015 12:52 PM IST
Bio Blast Pharma reports promising data on muscle wasting drug
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Bio Blast Pharma Ltd said its lead experimental drug showed signs of being effective in treating patients with a rare muscle wasting disorder, besides also hitting the main goals of safety and tolerability.

The company said the experimental drug, Cabaletta, hit multiple efficacy endpoints during a mid-stage trial on patients suffering from oculopharyngeal muscular dystrophy, according to interim results from study.

Oculopharyngeal muscular dystrophy is a rare disease where patients develop swallowing difficulties, which could lead to death in severe cases.

Bio Blast said the drug improved patients' muscle function while also reducing difficulty in swallowing.

The company said it would discuss results from this study and a second mid-stage study with the U.S. Food and Drug Administration before it started a late-stage study.

Bio Blast pharma is also testing the drug as a treatment for spinocerebellar ataxia type 3, another genetic condition that affects the nerves in the brain.

(Reporting by Vidya L Nathan in Bengaluru; Editing by Savio D'Souza)
Meghna A Singhania
Meghna A Singhania

Meghna A Singhania is the founder and Editor-in-Chief at Medical Dialogues. An Economics graduate from Delhi University and a post graduate from London School of Economics and Political Science, her key research interest lies in health economics, and policy making in health and medical sector in the country. She is a member of the Association of Healthcare Journalists. She can be contacted at meghna@medicaldialogues.in. Contact no. 011-43720751

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