FDA grants orphan drug designation to myotonic dystrophy treatment

The FDA has granted orphan drug designation to an investigational therapeutic to treat myotonic dystrophy type 1, the most common form of muscular dystrophy in adults, according to its manufacturer.
According to a release from Juvena Therapeutics, JUV-161 is the company’s lead candidate of engineered biologics for multiple myopathies and metabolic diseases. Human trials of JUV-161, which possesses an IGF-2 protein developed for subcutaneous administration, are expected to begin this year.
“People living with DM1 deserve safe, effective, and rejuvenating treatments that can repair

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