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Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

XTalks

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company. Related: Hemgenix Approved as First Gene Therapy for Hemophilia B “This is a devastating disease,” said M.

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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.

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Sanofi wagers $400m on miRecule muscular dystrophy therapy

pharmaphorum

There’s little detail on the focus of the alliance, although TrialSpark’s main focus is on rheumatology, dermatology, central nervous system, and cardiometabolic diseases. The aim will be to identify promising candidates in areas of “unmet patient need.”

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