Victoria Gray spent 34 years battling the debilitating pain of sickle cell disease. Then she volunteered to be the world's ...

Sidra Medicine has marked a significant medical achievement by securing regulated access to sepiapterin for patients with Phe ...

DOHA: Sidra Medicine has announced a medical achievement by facilitating access to sepiapterin for patients with ...

Lipoprotein(a), a fatty particle, can clog arteries just as surely as cholesterol but often goes undetected, striking ...

Phase 1 proof-of-concept study of CLY-124 in Sickle Cell Disease expected in Q4 2026In vivo preclinical data assessing new compounds vs. standard of care in gold-standard, translatable myelofibrosis ...

JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR"), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced today that it will present at ...

Once-weekly TransCon® CNP and TransCon® hGH combination therapy showed durable growth without compromising safety or ...

GenEditBio Limited ("GenEditBio"), a clinical-stage biotechnology startup focusing on genome-editing therapeutic solutions, today announced that the U.S. Food and Drug Administration (FDA) has cleared ...

Tisento Therapeutics today announced that the global Phase 2b PRIZM study is fully enrolled. PRIZM is evaluating zagociguat – a once-daily, oral investigational medicine – as a treatment for the rare ...

As BioMendics prepares to share its progress during the 2026 J.P. Morgan Healthcare Conference and Biotech Showcase, the Ohio-based biotech is advancing what could become the first disease-modifying ...

Recludix Pharma, a clinical-stage company leading the discovery and development of inhibitors of challenging targets for inflammatory disease, today announced the expansion of its Scientific Advisory ...

Knowable Magazine reports 2025 faced turmoil in U.S. science amid job cuts and budget slashes, yet saw advances in gene ...