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Sarepta Announces FDA Acceptance of Golodirsen NDA for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53

CAMBRIDGE, Mass., Feb. 14, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced the Food and Drug Administration, Division of Neurology (the Division), has accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action …

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Ultragenyx and Kyowa Kirin Announce Positive Results for Crysvita from Phase 3 Study in Children with X-linked Hypophosphatemia

NOVATO, Calif., TOKYO and LONDON, Feb. 14, 2019 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, Kyowa Hakko Kirin Co. Ltd (Kyowa Hakko Kirin), and Kyowa Kirin International PLC (Kyowa Kirin International) today …

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Apellis’ APL-2 Receives Fast Track Designation from the FDA for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

WALTHAM Mass., and CRESTWOOD, Ky., Feb. 11, 2019 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals Inc., (Nasdaq:APLS) a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation …

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FDA Grants Clementia’s Palovarotene Rare Pediatric Disease Designation for Fibrodysplasia Ossificans Progressiva

MONTREAL, Feb. 11, 2019 (GLOBE NEWSWIRE) — Clementia Pharmaceuticals Inc. (Nasdaq: CMTA), a clinical-stage biopharmaceutical company innovating treatments for people with ultra-rare bone disorders and other diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to palovarotene for the treatment of fibrodysplasia …

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BioMarin’s Ongoing Study of Brineura Demonstrates Durable Treatment Benefit in Patients with CLN2 Disease

SAN RAFAEL, Calif., Feb. 7, 2019 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that an ongoing open-label extension study treating patients with Brineura® (cerliponase alfa) continued to show a reduced rate of decline compared to a natural history cohort of CLN2 disease for three years as measured by the CLN2 Clinical Rating …

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Sangamo Announces Interim Results Of Phase 1/2 Study of SB-913 Showing Preliminary Evidence Of In Vivo Genome Editing In Patients With MPS II

BRISBANE, Calif., Feb. 7, 2019 /PRNewswire/ — Sangamo Therapeutics, Inc. (NASDAQ: SGMO) today reported preliminary molecular and enzymatic evidence of editing of the human genome in vivo (inside the body). These findings are part of the interim results from the Phase 1/2 CHAMPIONS Study evaluating SB-913, a zinc finger nuclease (ZFN) in vivo genome editing product candidate for …

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Solid Biosciences Announces Preliminary SGT-001 Data and Intention to Dose Escalate in IGNITE DMD Clinical Trial for Duchenne Muscular Dystrophy

CAMBRIDGE, Mass., Feb. 07, 2019 (GLOBE NEWSWIRE) — Solid Biosciences (Nasdaq: SLDB) announced today preliminary findings from IGNITE DMD, the Company’s Phase I/II dose-ascending clinical trial evaluating the safety and efficacy of SGT-001 microdystrophin gene transfer for the treatment of Duchenne muscular dystrophy (DMD). Initial three-month biopsy data showed low …

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Sanofi Announces FDA Approval of Cablivi, the First Nanobody-Based Medicine for Patients with a Rare Blood-Clotting Disorder

PARIS – February 6, 2019 – The U.S. Food and Drug Administration (FDA) has approved Cablivi® (caplacizumab-yhdp) in combination with plasma exchange and immunosuppression for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in adults.  Cablivi is the first FDA approved therapy specifically indicated for the treatment of aTTP. “The U.S. approval …

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Data from Alnylam’s Phase 1 Trial of Givosiran Published in The New England Journal of Medicine

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that results from the Phase 1 study of givosiran, an investigational, subcutaneous RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP), were published online todayin The New England Journal of Medicine (NEJM). The …

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Zogenix Submits Fintepla Applications to the FDA and EMA for the Treatment of Dravet Syndrome

EMERYVILLE, Calif., Feb. 06, 2019 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ:ZGNX), a global pharmaceutical company developing rare disease therapies, today announced it has completed its rolling submission of a New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) and submitted a Marketing Authorization Application (MAA) to the …

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