The treatment for acquired thrombotic thrombocytopenic purpura, a rare blood disorder that causes blood clots in the small ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

“This partnership marks a major milestone in our mission to transform how rare disease therapies reach patients in Europe,” said Adam Plich, Founder and CEO of Avanzanite. “We provide biotech partners ...

The MarketWatch News Department was not involved in the creation of this content. -- Key presentations include two updates from a phase 3 trial evaluating investigational treatment with Mim8 ...

CAMBRIDGE, Mass., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients ...

– Both Mitapivat Dose Arms Achieved Statistically Significant Hemoglobin Response in Phase 2 Portion of RISE UP Pivotal Study in Sickle Cell Disease, Compared to the Placebo Arm; Data Support ...

In a major leap for genetic diagnostics, scientists from the University of Melbourne and the Murdoch Children's Research Institute (MCRI) have developed a groundbreaking rapid blood test that can ...

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ...

PYRUKYND® (mitapivat) is a first-in-class pyruvate kinase (PK) activator, approved by the European Commission for adults with PK deficiency The agreement includes commercialization and distribution ...

King Faisal Specialist Hospital & Research Centre continues to advance pediatric care through specialized programs dedicated to solid tumors and non-malignant blood disorders, supported by ...

A new "proteomic" blood test can accurately screen a newborn's DNA for thousands of rare, inherited diseases by analyzing just a single-milliliter sample, according to a study to be presented Monday.