An experimental, oral medicine originally developed by PTC Therapeutics and now licensed to Roche improved the muscle function of babies with a fatal form of the rare, genetic disease known as spinal muscular atrophy, according to preliminary results from a clinical trial presented Saturday.
More testing needs to be done to confirm these findings, but if they hold up, the new drug from PTC and Roche could be a significant competitive threat to Spinraza, the first chronic drug approved to treat SMA patients, marketed by Biogen.
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