Tag Archives: enzyme replacement therapy

October, 2018

8 October
FDA Approves Leadiant’s Revcovi for the Treatment of ADA-SCID in Pediatric and Adult Patients

GAITHERSBURG, Md.–(BUSINESS WIRE)–Leadiant Biosciences, Inc. today announced that the Food and Drug Administration (FDA) has granted approval to Revcovi™ (elapegademase-lvlr) injection in the U.S. Revcovi is a new enzyme replacement therapy (ERT) for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. “We are gratified …
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27 April
Centogene Publishes Study Identifying the Best Biomarker for Monitoring Response to Therapy for Patients with Gaucher Disease

ROSTOCK, Germany–(BUSINESS WIRE)–CENTOGENE, the worldwide leader in elucidating rare disease genetics for patients, clinicians and pharmaceutical partners, announced the publication of a scientific paper in the March issue of the American Journal of Hematology. Professor Ari Zimran, M.D., Gaucher unit, Shaare Zedek Medical Center, Hebrew University, Jerusalem, with additional colleagues from …
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8 June
Breakthrough Status Granted to Genzyme’s Rare Enzyme Replacement Drug

Genzyme, a Sanofi company, recently announced that its investigational enzyme-replacement therapy was granted Breakthrough Therapy designation from US health regulators. The company said that the agency granted the status to its olipudase alfa for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick …
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8 April
FDA Accepts ArmaGen’s IND for its Hurler Syndrome Candidate

Privately-held biotech ArmaGen, Inc. announced today that US health regulators have accepted for review its investigational New Drug (IND) application for its drug for a rare lysosomal storage disease. The company, focused on developing novel therapies to treat severe neurological disorders, said that the US Food and Drug Administration (FDA) …
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