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Rare Diseases

Sarepta Announces Positive Preliminary Results from Phase 1/2a Gene Therapy Trial in Duchenne Muscular Dystrophy

CAMBRIDGE, Mass., June 19, 2018 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from …

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FDA Lifts Clinical Hold on Solid Biosciences’ Clinical Trial for its Investigational Duchenne Muscular Dystrophy Treatment

CAMBRIDGE, Mass., June 18, 2018 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (NASDAQ:SLDB) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001, for the treatment of Duchenne muscular dystrophy (DMD). …

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Blueprint Avapritinib Demonstrates Profound and Durable Clinical Activity in Advanced Systemic Mastocytosis Trial

CAMBRIDGE, Mass., June 15, 2018 /PRNewswire/ — Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, today announced updated data from its ongoing Phase 1 EXPLORER clinical trial of avapritinib, a potent and highly selective KIT and PDGFRA inhibitor in development for patients with …

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Novartis Survey Uncovers Real-World Impact of Immune Thrombocytopenia on Patients’ Quality of Life

Basel, June 15, 2018 – Many patients with the rare blood disorder immune thrombocytopenia (ITP) find the disease has a negative impact on their everyday quality of life, according to interim results of a Novartis survey, called I-WISh, presented today at the 23rd Congress of the European Hematology Association (EHA) in Stockholm, Sweden …

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FDA Approves Genentech’s Rituxan (Rituximab) for Pemphigus Vulgaris

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Rituxan®(rituximab) for the treatment of adults with moderate to severe pemphigus vulgaris (PV), a rare, serious, potentially life-threatening condition characterized by progressive painful blistering of the …

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Alnylam Reports Updated Positive Results from Phase 1/2 Study of Lumasiran in Primary Hyperoxaluria Type 1

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today new positive results from its Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1). Results were presented at the OxalEurope, European Hyperoxaluria Consortium, taking …

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FDA Grants Orphan Drug Designation to Ironwood’s Olinciguat for the Treatment of Sickle Cell Disease

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Ironwood Pharmaceuticals, Inc. (NASDAQ: IRWD), a commercial biotech company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to olinciguat (IW-1701) for the treatment of patients with sickle cell disease. Olinciguat is an orally administered soluble guanylate cyclase (sGC) stimulator. “There is an …

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FDA Grants Orphan Drug Designation to Alnylam’s Investigational RNAi Therapeutic for Treatment of Transthyretin-Mediated Amyloidosis

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to ALN-TTRsc02, an investigational RNAi therapeutic for the treatment of transthyretin-mediated amyloidosis. ALN-TTRsc02 has the potential to be a once-quarterly, low volume, subcutaneously …

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Centogene and Aldeyra Therapeutics Partner to Advance Novel Therapeutics for Patients with Sjögren-Larsson Syndrome

ROSTOCK, Germany–(BUSINESS WIRE)–CENTOGENE, the worldwide leader in elucidating rare disease genetics for patients, clinicians and pharmaceutical partners, today announced that it has entered into an agreement with Aldeyra Therapeutics Inc., a biotechnology company developing next-generation medicines to improve the lives of patients with immune-mediated diseases. Under the terms of the agreement, …

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FDA Approves Biomarin’s Palynziq Injection for Treatment of Phenylketonuria, A Rare Genetic Disease

SAN RAFAEL, Calif., May 24, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that BioMarin received standard approval from the U.S. Food and Drug Administration (FDA) for Palynziq™ (pegvaliase-pqpz) Injection to reduce blood phenylalanine (Phe) concentrations in adult patients with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 micromol/L …

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