Tag Archives: ERT

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 December
FDA Approves Alexion’s Kanuma for Patients with a Rare Enzyme Disease

CHESHIRE, Conn.–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) announced today that the U.S. Food and Drug Administration (FDA) has approved Kanuma™(sebelipase alfa) for the treatment of patients of all ages with a diagnosis of lysosomal acid lipase deficiency (LAL-D). Kanuma, an innovative enzyme replacement therapy (ERT), is the first therapy approved …
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