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MANF receives orphan drug designation for retinitis pigmentosa
MANF, an investigational drug, has received orphan drug designation for the treatment of retinitis pigmentosa from the U.S. Food and Drug Administration, Amarantus BioScience Holdings announced in a press release.Preclinical data showed that MANF (mesencephalic astrocyte-derived neurotrophic factor) had a protective functional effect in an animal model of retinitis pigmentosa, and toxicology studies showed a single intravitreal administration of a therapeutically relevant dose was well-tolerated.