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January 27, 2026 08:30 ET | Source: Quoin Pharmaceuticals, Inc.

Quoin Pharmaceuticals Announces Submission to Japanese MHLW for Orphan Drug Designation for QRX003 in Netherton Syndrome

Confirmation Received from MHLW That QRX003 Qualifies for Both Orphan Drug Designation and Fast Track Regulatory Review Status Quoin Initiating the Establishment of a Japanese Subsidiary to...
January 26, 2026 09:15 ET | Source: Longeveron

Longeveron Announces FDA Grants Type C Meeting Ahead of Data Readout for Pivotal Phase 2 Clinical Trial (ELPIS II) Evaluating Treatment for Hypoplastic Left Heart Syndrome (HLHS)

Longeveron granted FDA Type C meeting ahead of Q3 2026 data readout of pivotal clinical trial for stem cell therapy in hypoplastic left heart syndrome.
January 26, 2026 07:00 ET | Source: Agios Pharmaceuticals, Inc.

Agios to Host Fourth Quarter and Full Year 2025 Financial Results Conference Call and Webcast on February 12 at 8:00 a.m. ET

Agios announced the company will host a conference call and live webcast on Thursday, February 12, 2026, at 8:00 a.m. ET to report its 4Q and full year
January 16, 2026 10:50 ET | Source: Baylor Genetics

Baylor Genetics Supports Bipartisan House Bill to Broaden Medicaid Access to Genomic Sequencing for Children who Meet Medical Criteria

HOUSTON, Jan. 16, 2026 (GLOBE NEWSWIRE) -- Baylor Genetics, a clinical diagnostic laboratory at the forefront of genetic testing, announced its support for the Genomic Answers for Children’s Health...
January 14, 2026 09:00 ET | Source: Muscular Dystrophy Association

Muscular Dystrophy Association’s Legacy Awards Honor Innovators in Clinical and Scientific Research, and Community Impact at the 2026 MDA Clinical & Scientific Conference in March

New York, Jan. 14, 2026 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) is proud to announce the recipients of the 2026 MDA Legacy Awards, recognizing excellence in scientific and...
January 13, 2026 07:45 ET | Source: Fortress Biotech, Inc.

Fortress Biotech and Cyprium Therapeutics Announce U.S. FDA Approval of ZYCUBO® (copper histidinate), the First and Only Approved Treatment for Menkes Disease in the United States

Rare Pediatric Disease Priority Review Voucher (PRV) granted by FDA at approval to be transferred from Sentynl Therapeutics to Cyprium Cyprium eligible to receive tiered royalties and up to $129...
January 12, 2026 11:05 ET | Source: Biogen Inc.

Biogen Receives European Commission Approval for High Dose Regimen of SPINRAZA® (nusinersen) for Spinal Muscular Atrophy

Approval is supported by data from the DEVOTE study which showed the benefit of the SPINRAZA 50 mg and 28 mg regimen in both treatment-naïve and previously-treated nusinersen patients with SMA1Biogen...
January 12, 2026 07:00 ET | Source: Agios Pharmaceuticals, Inc.

Agios Outlines 2026 Strategic Priorities and Key Milestones to Accelerate Rare Disease Portfolio Growth

Agios today announced its 2026 strategic priorities and key milestones anticipated during the year.
January 08, 2026 08:03 ET | Source: Catalyst Pharmaceuticals, Inc.

Catalyst Pharmaceuticals Receives Prestigious Ranking on Forbes 2026 List of America's Most Successful Small-Cap Companies

CORAL GABLES, Fla., Jan. 08, 2026 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing,...
January 08, 2026 07:00 ET | Source: Tisento Therapeutics Inc

Tisento Therapeutics Completes Enrollment in PRIZM, a Global Phase 2b Study of Zagociguat for the Treatment of MELAS

Tisento's global Phase 2b PRIZM study, which is evaluating zagociguat for the treatment of the rare mitochondrial disease MELAS, is fully enrolled