Remove Dermatology Remove Genetic Disease Remove Genetics
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Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

XTalks

Vyjuvek is also the first drug approved to treat the disease and is Krystal’s first approved product. DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis.

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Sanofi wagers $400m on miRecule muscular dystrophy therapy

pharmaphorum

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. The aim will be to identify promising candidates in areas of “unmet patient need.”

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Spevigo Becomes First Treatment Option for Generalized Pustular Psoriasis Flares

XTalks

GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it. Waldman Department of Dermatology. Longer trials are needed to fully understand the effects and safety of Spevigo in those affected by GPP using the treatment long-term.