Aardvark Therapeutics Voluntarily Pauses Phase 3 HERO Trial of ARD-101 in Prader-Willi Syndrome

Aardvark Therapeutics, Inc. (Aardvark) (Nasdaq: AARD), a clinical-stage biopharmaceutical company focused on developing novel, small-molecule therapeutic...

March 02, 2026 | Monday | News
Savara Advances MOLBREEVI Toward Approval with FDA Priority Review and 2026 Decision Timeline

Priority Review Granted, Prescription Drug User Fee Act (PDUFA) Target Action Date Set for August 22, 2026 -- -- Company Plans to Submit the MOLBREEV...

February 24, 2026 | Tuesday | News
Andelyn Biosciences Expands Commitment to Rare Disease Community with NKH Gene Therapy Manufacturing Program

Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been selecte...

February 19, 2026 | Thursday | News
Konovo Acquires Rare Patient Voice to Deepen Access to Hard-to-Reach Rare Disease Communities

Rare Patient Voice’s 200,000+ registered patients and caregivers expand Konovo’s ability to deliver connected insights across a broad range o...

February 09, 2026 | Monday | News
Pfizer CEO Albert Bourla Signals Confidence in 2026 as Company Delivers Solid 2025 Results

Dr. Albert Bourla, Chairman and CEO of Pfizer, said: “With excellent execution in 2025, we delivered a solid financial performance and strengt...

February 05, 2026 | Thursday | Company results
Antibodies Inc. Launches Recombinant Guinea Pig RBPMS Antibody to Advance Retinal Ganglion Cell Research

Antibodies Inc., a leading supplier of validated life science research reagents,  announced the availability of its Recombinant Guinea Pig RBPMS An...

January 23, 2026 | Friday | News
Gates Foundation, Novo Nordisk Foundation, and Wellcome Launch $60M Global Consortium to Accelerate Antibiotic Discovery Against AMR

The Gates Foundation, Novo Nordisk Foundation, and Wellcome today awarded a total of $60 million in new grant funding over the next three years to resear...

January 23, 2026 | Friday | News
Charles River Laboratories International And Gazi University Faculty of Medicine Advance Rare Disease Gene Therapy Through Strategic CDMO Collaboration

Charles River Laboratories International, Inc. (NYSE: CRL) and Gazi University Faculty of Medicine, announced a gene therapy contract developme...

January 22, 2026 | Thursday | News
Atossa Therapeutics Secures FDA Orphan Drug Designation for (Z)-Endoxifen in Duchenne Muscular Dystrophy

Designation further supports (Z)-Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pedi...

January 19, 2026 | Monday | News
China Approves Sanofi’s Myqorzo and Redemplo for Cardiovascular and Rare Metabolic Diseases

Approval of Myqorzo for obstructive hypertrophic cardiomyopathy and Redemplo for familial chylomicronemia syndrome Underscores Sanofi’s long-ter...

January 16, 2026 | Friday | News
IDT Relocates Bay Area Operations to Expanded Sunnyvale Innovation Hub

Genomics leader relocates to an expanded footprint in Sunnyvale New location enables customer continuity and provides increased innovation space to driv...

January 15, 2026 | Thursday | News
HUTCHMED’s Sovleplenib Meets Primary Endpoint in Phase III ESLIM-02 Trial in wAIHA

— Delivers rapid, durable responses in wAIHA, the more common form of this potentially life-threatening disease —  HUTCHMED (China) Lim...

January 08, 2026 | Thursday | News
Zevra Therapeutics Signs Exclusive Expanded Access Distribution Deal With Uniphar for MIPLYFFA

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare ...

December 31, 2025 | Wednesday | News
SynaptixBio Backs FDA Priority Review Voucher Renewal as Critical Lifeline for Rare Disease Innovation

SynaptixBio, the only company licensed to commercialise a treatment for a rare, deadly disease, says the Priority Review Voucher scheme is a lifeline for...

December 22, 2025 | Monday | News

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