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July 08, 2026 05:08 ET | Source: A Race Against Blindness

Nonprofit Launches One of the Nation’s Most Comprehensive Education Resources for Inherited Retinal Diseases

Nonprofit Launches One of the Nation’s Most Comprehensive Education Resources for Inherited Retinal Diseases
July 07, 2026 07:00 ET | Source: Agios Pharmaceuticals, Inc.

U.S. FDA Grants Priority Review to Agios’ sNDA for Mitapivat in Sickle Cell Disease

Agios announces that the FDA has accepted its sNDA for mitapivat in sickle cell disease with a Priority Review
July 07, 2026 05:00 ET | Source: Italfarmaco

Presentation of New Clinical Data on Givinostat in Duchenne Muscular Dystrophy at ICNMD 2026

Phase 3 EPIDYS MRI data quantify givinostat’s potential to preserve functional muscle tissue and reduce fat infiltration in patients with Duchenne muscular dystrophy1Updated open-label extension data...
July 01, 2026 08:51 ET | Source: SERB Pharmaceuticals

SERB Pharmaceuticals Completes Acquisition of European and MENA Rights to Idefirix® (imlifidase) from Hansa Biopharma

SERB Pharmaceuticals has completed the acquisition of development and commercialization rights to Idefirix (imlifidase) in Europe
July 01, 2026 03:00 ET | Source: Oxford-Harrington Rare Disease Centre

Oxford-Harrington Rare Disease Centre Strengthens Rare Childhood Cancer Programme Through Appointment of New Little Princess Trust Professor of Paediatric Oncology

Professor Isidro Cortés-Ciriano, an internationally recognised scientist with extensive experience in childhood cancer research, will accelerate Oxford-Harrington’s mission to translate discoveries...
June 30, 2026 08:03 ET | Source: Catalyst Pharmaceuticals, Inc.

Vamorolone Demonstrates On-Target Glucocorticoid Activity Without the Immunosuppression Characteristic of Traditional Corticosteroids at Clinical Doses

CORAL GABLES, Fla., June 30, 2026 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing,...
June 29, 2026 07:00 ET | Source: Agios Pharmaceuticals, Inc.

Agios to Host Second Quarter 2026 Financial Results Conference Call and Webcast on July 30 at 8:00 a.m. ET

Agios will report its second quarter 2026 financial results and business highlights on Thursday, July 30, 2026.
June 22, 2026 09:30 ET | Source: CurePSP

CurePSP Expands Grant Program with $155,000 Investment in Studying Care for Rare Neurological Diseases

NEW YORK, June 22, 2026 (GLOBE NEWSWIRE) -- CurePSP announced the latest recipients of the Collaborative Approaches to Resources, Education and Support (CARES) grants, awarding a total of $155,000...
June 18, 2026 14:43 ET | Source: Constantiam Biosciences

Constantiam Biosciences and Cincinnati Children’s Collaborate to Accelerate First-in-Class Neuronopathic Gaucher Disease Therapies

WATERTOWN, Mass. and CINCINNATI, June 18, 2026 (GLOBE NEWSWIRE) -- Constantiam Biosciences and Cincinnati Children's today announced a strategic collaboration through an exclusive option for future...
June 18, 2026 07:00 ET | Source: Essential Pharma

Essential Pharma appoints Becki Morison as Chief Executive Officer

Pharmaceutical executive brings 25+ years of international commercial leadership and rare disease experience Egham, UK – 18 June 2026 – Essential Pharma ("Essential" or "the Company"), a global...