COMBINEDBrain Received the Global Genes 2022 Health Equity RARE Patient Impact Grant
September 29, 2022 11:27 ET | COMBINEDBrain Inc
NASHVILLE, Tenn., Sept. 29, 2022 (GLOBE NEWSWIRE) -- COMBINEDBrain (Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders), a non-profit organization of patient advocacy...
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The New England Journal of Medicine Publishes Pivotal Tofersen Data that Show Benefits in Rare, Genetic Form of ALS
September 21, 2022 17:15 ET | Biogen Inc.
Publication includes the Phase 3 VALOR SOD1-ALS trial and its open-label-extension study, underscoring the importance of these longer-term data12-month data show earlier initiation of tofersen slowed...
Elixiron Immunotherapeutics Announces Orphan Drug Designation Granted to Interferon-Gamma-Neutralizing Antibody, EI-001 for the Treatment of Hemophagocytic Lymphohistiocytosis
September 06, 2022 10:00 ET | Elixiron Immunotherapeutics
SAN FRANCISCO, Sept. 06, 2022 (GLOBE NEWSWIRE) -- Elixiron Immunotherapeutics, a clinical stage biotech company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan...
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Travere Therapeutics and CSL Vifor Announce EMA has Accepted for Review the Conditional Marketing Authorization Application for Sparsentan for the Treatment of IgA Nephropathy
August 22, 2022 07:00 ET | Travere Therapeutics, Inc.
A review decision by the European Medicines Agency (EMA) is expected in the second half of 2023 If approved, sparsentan will be a first-in-class treatment to address the significant unmet medical...
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Travere Therapeutics Reports Second Quarter 2022 Financial Results
August 04, 2022 07:00 ET | Travere Therapeutics, Inc.
SAN DIEGO, Aug. 04, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today reported its second quarter 2022 financial results and provided a corporate update. New Drug Application...
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Travere Therapeutics Provides Regulatory Updates on its Development Programs
August 03, 2022 16:05 ET | Travere Therapeutics, Inc.
PDUFA target action date for NDA for sparsentan in IgAN on-track for November 17, 2022 Company to utilize traditional approval process for sparsentan in FSGS following completion of the pivotal Phase...
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Frontera Therapeutics Completes $160 Million Series B Financing to Fund Clinical Development and Manufacturing Capabilities
July 19, 2022 08:00 ET | Frontera Therapeutics
FDA clears Investigational New Drug (IND) application for lead gene therapy product candidate, FT-001, for the treatment of a rare inherited eye disease leading to severe visual impairment that may...
ANGEL AID Unveils Raregivers™ Global Mental Health Initiative for Caregivers, Patients and Professionals
July 12, 2022 11:00 ET | ANGEL AID
LOS ANGELES, July 12, 2022 (GLOBE NEWSWIRE) -- ANGEL AID, a nonprofit support organization for families of children with rare diseases, today announced the Raregivers™ Coalition Mental Health &...
Insilico Medicine identified multiple new targets for amyotrophic lateral sclerosis (ALS) using its AI-driven target discovery engine
July 07, 2022 09:00 ET | InSilico Medicine
New York, July 07, 2022 (GLOBE NEWSWIRE) -- Insilico Medicine, a clinical-stage end-to-end artificial intelligence (AI)-driven drug discovery company, announced today that the company has identified...
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Global Orphan Designated Rare Cancer Drug Market Forecast 2028
July 01, 2022 06:50 ET | KuicK Research
Singapore, July 01, 2022 (GLOBE NEWSWIRE) -- Global Orphan Cancer Drug Market, Drug Sales, Price & Clinical Trials Insight 2028 Report Highlights: Global Orphan Designated Market...