Orphan Drug Designation for Myostatin Inhibitor for Muscular Dystrophy
Milo Biotechnology announced that AAV1-FS344 has been granted Orphan Drug designation from the FDA for the treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.
The program is currently in a Phase 1/2 trial in adult patients with Becker muscular dystrophy and inclusion body myositis.
Duchenne muscular dystrophy is the most common inherited myopathy, caused by the absence of dystrophin protein. Becker muscular dystrophy, a less severe form of Duchenne muscular dystrophy, is caused by reduced levels of dystrophin protein. With both types, patients experience progressive muscle weakness, and cardiac and respiratory complications.
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