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...
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...
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...
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...
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...
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...
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...