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