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...
CENTOGENE and Agios Expand Partnership for Clinical Development of PYRUKYND® (mitapivat) to Treat Children With Rare Blood Disease
June 13, 2022 06:30 ET | Centogene NV
Expanded collaboration to include Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies CENTOGENE’s genetic testing and Biodatabank identify causative mutations, including the UGT1A1 and PKLR genes, and...
Ultragenyx Announces Positive Longer-term Durability Data from Two Phase 1/2 Gene Therapy Studies at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
May 19, 2022 08:00 ET | Ultragenyx Pharmaceutical Inc.
Durable and clinically meaningful responses sustained for more than three years post-treatment in study of DTX401 for GSDIa and four or more years in study of DTX301 for OTC NOVATO, Calif., May ...
Ultragenyx Acquires Global Rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics
May 17, 2022 08:00 ET | Ultragenyx Pharmaceutical Inc.
ABO-102 / UX111 is being evaluated in ongoing pivotal Transpher A trial in patients with MPS IIIA Interim data featured in encore oral presentation at American Society of Gene & Cell Therapy...
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Decibel Therapeutics Reports First Quarter 2022 Financial Results and Corporate Update
May 12, 2022 07:30 ET | Decibel Therapeutics, Inc.
- Multiple milestones anticipated for 2022 including submission of IND and/or CTA for DB-OTO, top-line results from interim analysis of Phase 1b trial of DB-020 for cisplatin-induced hearing loss and...