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Rare Diseases

AbbVie’s Humira Gains FDA Approval to Treat Patients with Certain Types of Uveitis

NORTH CHICAGO, Ill., June 30, 2016 /PRNewswire/ — AbbVie (NYSE: ABBV), a global biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has approved HUMIRA® (adalimumab) for the treatment of non-infectious intermediate, posterior and panuveitis. HUMIRA is now the first and only FDA-approved non-corticosteroid therapy available for …

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GW Pharma’s Epidiolex Succeeds in Late-Stage Lennox-Gastaut Syndrome Study

LONDON, June 27, 2016 (GLOBE NEWSWIRE) — GW Pharmaceuticals plc (Nasdaq:GWPH) (AIM:GWP) (“GW,” “the Company” or “the Group”), a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, announces positive results of the first randomized, double-blind, placebo-controlled Phase 3 clinical trial of its …

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FDA Clears Sangamo Biosciences’ IND for ZFN-Mediate Genome Editing Treatment of MPS II

RICHMOND, Calif., June 20, 2016 /PRNewswire/ — Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug application (IND) for SB-913, a zinc finger nuclease (ZFN)-mediated approach designed as a single treatment with …

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Marathon Seeks FDA Approval for its Duchenne Muscular Dystrophy Drug

NORTHBROOK, Ill.–(BUSINESS WIRE)–Marathon Pharmaceuticals, LLC, a biopharmaceutical company developing treatments for rare diseases, today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug deflazacort for the treatment of patients with Duchenne muscular dystrophy (DMD), the most common and …

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FDA Grants Breakthrough Therapy Designation for Two of Shire’s Rare GI Drugs

Lexington, Mass. – June 13, 2016 – Shire plc (LSE: SHP, NASDAQ: SHPG) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for two investigational products for rare diseases: SHP621 (budesonide oral suspension, or BOS) for eosinophilic esophagitis (EoE), and SHP625 (maralixibat) for progressive …

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Ultragenyx and Takeda Partner for Development Treatments for Rare Genetic Diseases

NOVATO, Calif. and OSAKA, Japan, June 07, 2016 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare genetic diseases, and Takeda Pharmaceutical Company Limited (TSE: 4502), today announced a strategic partnership to develop and commercialize therapies to treat …

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FDA Requests Dystrophin Data from Sarepta Prior to Making a Decision on Eteplirsen NDA

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jun. 6, 2016– Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has requested that Sarepta provide dystrophin data, as measured by western blot, from biopsies already obtained from the ongoing confirmatory study of eteplirsen (PROMOVI), as …

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Biogen Collaborates with University of Pennsylvania to Advance Gene Therapy and Gene Editing Technologies

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biogen (NASDAQ:BIIB) today announced a broad collaboration and alliance with the University of Pennsylvania (“Penn”) to advance gene therapy and gene editing technologies. The expansive research and translational development collaboration has multiple objectives, but will primarily focus on the development of therapeutic approaches that target the eye, skeletal muscle and the central nervous system …

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WAVE Life Sciences to Advance Next-Generation Nucleic Acid Therapies to Address Unmet Need in DMD

CAMBRIDGE, Mass.–(BUSINESS WIRE)–WAVE Life Sciences Ltd. (NASDAQ:WVE), a genetic medicines company focused on developing stereopure nucleic acid therapies for patients impacted by rare diseases, today reaffirmed its commitment to advance next-generation nucleic acid therapies to address the significant unmet need of patients diagnosed with Duchenne Muscular Dystrophy (DMD). WAVE’s DMD …

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Collaborative Study of Whole Exome Sequencing Offers New Hope for Children with White Matter Disorders

SAN DIEGO–(BUSINESS WIRE)–Illumina, Inc. (NASDAQ: ILMN), Children’s National Health System and The University of Queensland (UQ) announced today that a study using whole exome sequencing (WES), a method to look at all the genes in the genome at once, yielded clinical diagnoses for 42 percent of patients with white matter abnormalities …

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