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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.
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.”
GPP occurs in individuals with specific gene variations as it is a geneticdisease , 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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