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A total of 135 posts are filed under Business
Sigma-Tau Pharmaceuticals, Inc. Changes Name to Leadiant Biosciences, Inc. And Reaffirms its Commitment to Rare Disease Communities
Sigma-Tau Pharmaceuticals, Inc., a leader in the development and commercialization of medicines for patients with rare diseases, today… Continue Reading
Sanofi Genzyme’s Phase 2 Clinical Trial to Evaluate Oral Substrate Reduction Therapy for Gaucher Disease Type 3
Sanofi Genzyme is investigating an orally administered substrate reduction therapy in Gaucher disease type 3 as part of the 2-part LEAP… Continue Reading
FDA Grants Priority Review Designation for Actemra in Giant Cell Arteritis
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has… Continue Reading
Fierce Biotech: Ex-GSK rare disease head moves to Genenta
by Ben Adams (source) Carlo Russo has joined a growing list of ex-GlaxoSmithKline staffers moving into biotech after working on and helping… Continue Reading
Lysogene Receives Rare Pediatric Disease Designation from FDA for LYS-GM101 for Treatment of GM1 Gangliosidosis
Lysogene, a leading, clinical-stage biotechnology company specializing in gene therapy for rare central nervous system diseases, today… Continue Reading
Why Health Insurers Won’t Cover this $300,000-a-year Rare Disease Drug
(Source) — Alison Willis Hoke’s twin sons were among the 12 boys in a key clinical trial that helped get Sarepta Therapeutics Inc.’s… Continue Reading
Medgadget: Future of Global Rare Disease Treatment Market by 2016 – 2024
It is estimated that, approximately 5% of the global population suffer from a rare disease and half of the global population affected by… Continue Reading