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Rare Diseases

Fibrocell and Castle Creek Pharmaceuticals Collaborate for Development and Commercialization of FCX-007 Gene Therapy

EXTON, Pa., April 15, 2019 (GLOBE NEWSWIRE) — Fibrocell Science, Inc. (Nasdaq: FCSC), a cell and gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, today announced the completion of a collaboration agreement with Castle Creek Pharmaceuticals to develop and commercialize Fibrocell’s lead gene …

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Zogenix Receives Refusal to File Letter from the FDA for its Fintepla NDA for Treatment of Seizures Associated with Dravet Syndrome

EMERYVILLE, Calif., April 08, 2019 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ: ZGNX), a global pharmaceutical company developing rare disease therapies, today announced that it received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for FINTEPLA® (ZX008, fenfluramine hydrochloride) for …

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Celgene and Acceleron Submit Luspatercept BLA to the FDA for Myelodysplastic Syndromes and Beta-Thalassemia Indications

SUMMIT, N.J. & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced that Celgene has submitted a Biologics License Application (BLA) for luspatercept, an erythroid maturation agent, for the treatment of adult patients with very low to intermediate risk myelodysplastic syndromes (MDS)-associated anemia who have …

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Dystrogen Therapeutics Announces That Gene Therapy Successfully Cuts Off Production of Neuron-Destroying Protein in Huntington’s Disease

CHICAGO, April 5, 2019 /PRNewswire/ — Scientist from Dystrogen Therapeutics Corp. will present data supporting a potential cutting-edge therapy for neurodegenerative diseases caused by trinucleotide repeats, such as Huntington’s disease (HD) and spinocerebellar ataxias (SCAs). Early data confirmed that the therapy has the ability to selectively silence only the mutated …

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Acceleron Discontinues Development of its Phase 1 Neuromuscular Disorders Molecule ACE-2494

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced it is discontinuing development of ACE-2494, a systemic muscle agent the company had been studying in a Phase 1 healthy volunteer trial for …

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Abeona Therapeutics Receives FDA Fast Track Designation for ABO-101 for Treatment of Sanfilippo Syndrome Type B (MPS IIIB)

NEW YORK and CLEVELAND, April 04, 2019 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in cell and gene therapy development, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ABO-101, the Company’s novel one-time gene therapy for Sanfilippo syndrome …

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FDA Approves ADMA Biologics’ Asceniv, a Novel Intravenous Immune Globulin

RAMSEY, N.J. and BOCA RATON, Fla., April 01, 2019 (GLOBE NEWSWIRE) — ADMA Biologics, Inc. (NASDAQ: ADMA) (“ADMA” or the “Company”), a vertically integrated commercial biopharmaceutical and specialty immunoglobulin company that manufactures, markets and develops plasma-derived biologics for the treatment of immune deficiencies and the prevention of certain infectious diseases, …

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Sarepta Therapeutics Announces Positive Expression Results from the Casimersen Arm of the Phase 3 ESSENCE Study in DMD Patients

CAMBRIDGE, Mass., March 28, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced results from its interim analysis of muscle biopsy endpoints comparing casimersen treatment to placebo in the ESSENCE study, also known as study 4045-301. ESSENCE is a global, …

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Proteostasis Announces Broad New Dataset from Proprietary Combination and Add-On CFTR Modulator Studies in Cystic Fibrosis

BOSTON, March 25, 2019 /PRNewswire/ — Proteostasis Therapeutics, Inc. (NASDAQ: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced results from Phase 1 clinical studies of its proprietary cystic …

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Enzyvant’s Investigational Farber Disease Enzyme Replacement Therapy Receives FDA Fast Track and Rare Pediatric Disease Designations

CAMBRIDGE, Mass. and BASEL, Switzerland, March 21, 2019 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing and commercializing transformative therapies for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease and Fast Track designations for RVT-801, an investigational enzyme …

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