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A total of 131 posts are filed under Business
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
Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-201 Gene Therapy Program in Juvenile Batten Disease
  Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical-stage biopharmaceutical company focused on developing therapies for… Continue Reading
SPINRAZA™ (nusinersen) Approved in U.S. to Treat Broad Range of Patients with Spinal Muscular Atrophy
– First Treatment Approved for SMA –– SPINRAZA Improved Motor Function in Patients with SMA; Greater Percentage of Infantile-Onset… Continue Reading
Arcturus Therapeutics Announces Collaboration with Takeda Pharmaceuticals to Develop RNA–based Therapeutics for the Treatment of Nonalcoholic Steatohepatitis (NASH)
San Diego, Calif., Dec 6, 2016 – Arcturus Therapeutics, Inc. (“Arcturus” or the “Company”), a leading RNA medicines company,… Continue Reading
Ultragenyx Gives Arcturus Rocket Shot in Potential $1.57B Deal
By Marie Powers, News Editor (Source) The up front is only $10 million, but Arcturus Therapeutics Inc. stands to collect up to $1.56 billion… Continue Reading

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