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Biogen to Acquire Nightstar Therapeutics Gaining Pipeline of Gene Therapy Candidates in Ophthalmology

CAMBRIDGE, Mass., March 04, 2019 (GLOBE NEWSWIRE) — Biogen (Nasdaq: BIIB) today announced that it has entered into an agreement to acquire Nightstar Therapeutics (NST; Nasdaq: NITE), a clinical-stage gene therapy company based in London, United Kingdom, which is focused on adeno-associated virus (AAV) treatments for inherited retinal disorders. Under …

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Clinigen and Eiger Launch Worldwide Managed Access Program for Lonafarnib for Patients with Progeria and Progeroid Laminopathies

BURTON UPON TRENT, England–(BUSINESS WIRE)–Clinigen Group plc (AIM:CLIN, ‘Clinigen’), the global pharmaceutical and services company, has partnered with Eiger BioPharmaceuticals, Inc. (NASDAQ:EIGR, ‘Eiger’) to launch a worldwide lonafarnib Managed Access Program for patients with Progeria and Progeroid Laminopathies. Progeria, also known as Hutchinson-Gilford Progeria Syndrome, is a rare and fatal …

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Sarepta Exercises Option to Acquire Myonexus Therapeutics and its Gene Therapy Candidates

CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has exercised its option to acquire Myonexus Therapeutics, a clinical-stage biotechnology company developing transformative gene therapies for five LGMDs: LGMD2E, LGMD2D, LGMD2B, LGMD2C and LGMD2L. Subject to satisfaction of …

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Horizon’s Phase 3 Trial Evaluating Teprotumumab in Active Thyroid Eye Disease Met Primary and All Secondary Endpoints

DUBLIN–(BUSINESS WIRE)–Horizon Pharma plc (Nasdaq: HZNP) today announced topline results from its Phase 3 confirmatory trial evaluating teprotumumab for the treatment of active thyroid eye disease (TED). The study met its primary endpoint, showing more patients treated with teprotumumab compared with placebo had a meaningful improvement in proptosis, or bulging …

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Sarepta Announces Positive and Robust Expression and Biomarker Data from the MYO-101 Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E, or Beta-Sarcoglycanopathy

CAMBRIDGE, Mass., Feb. 27, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from three Limb-girdle muscular dystrophy (LGMD) Type 2E clinical trial participants who received MYO-101. MYO-101 is a novel gene therapy intended to transduce skeletal and …

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Ipsen to Acquire Clementia Pharmaceuticals to Significantly Boost Rare Disease Portfolio

PARIS & MONTRÉAL–(BUSINESS WIRE)–Regulatory News: Ipsen (Euronext: IPN; ADR: IPSEY) and Clementia Pharmaceuticals (NASDAQ: CMTA) today announced that they have entered into an agreement for Ipsen to acquire Clementia Pharmaceuticals, including its key late-stage clinical asset palovarotene, an investigational retinoic acid receptor gamma (RARγ) selective agonist, for the treatment of …

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FDA Grants Breakthrough Therapy Designation to Amicus’ Investigational Pompe Disease Therapy

CRANBURY, N.J., Feb. 25, 2019 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has granted to Amicus a Breakthrough Therapy Designation (“BTD”) to AT-GAA for the treatment of late onset Pompe disease, an inherited lysosomal storage disorder caused by the deficiency of an enzyme known as …

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Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

NOVATO, Calif., Feb. 21, 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, today announced positive longer-term top-line safety and efficacy data from the first, lowest dose cohort of the ongoing Phase 1/2 …

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Takeda, Microsoft and EURORDIS Release Report with Action Plan to Help Rare Disease Community Shorten the Diagnostic Odyssey

NEW YORK and BRUSSELS, Feb. 20, 2019 /PRNewswire/ — The Global Commission to End the Diagnostic Odyssey for Children (“the Global Commission”), co-chaired by Takeda, Microsoft and EURORDIS-Rare Diseases Europe, an alliance of more than 800 rare disease patient organisations, today announced its actionable recommendations in a report to address …

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Cerecor Receives Fast Track Designation from FDA for CERC-801 for the Treatment of PGM1 Deficiency

ROCKVILLE, Md., Feb. 19, 2019 (GLOBE NEWSWIRE) — Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of treatments for rare and orphan diseases in pediatrics and neurology, announced today that the U.S. Food and Drug Administration (FDA) has designated Fast Track Designation …

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