Last month, the NICE endorsed Casgevy for use in the NHS under a managed access scheme to treat sickle cell disease.

Physicians and their patients report that engagement in an HIV-based community rivals drug effectiveness in the ... maps out potential ways to approach a curative strategy, and comments on the ...

Observed annually on the last day of February, Rare Disease Day shines a light on the challenges patients face and the need for stronger policies. Let’s understand what rare diseases are and how India ...

In the latest in our series dispelling common misconceptions in healthcare, Dr Peter Bagshaw discusses how psychological ...

25, 2025 — A bone marrow transplant process is safe and curative for adults with sickle ... Brain Scan Predicts Effectiveness of Spinal Cord Surgery Dec. 3, 2024 — A 10-minute brain scan ...

Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies ...

Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in ...

Sickle cell disease (SCD), the most common inherited blood disorder, affects millions worldwide. In the United States alone, ...

BioCryst Pharmaceuticals, a rare disease biotech, shows strong revenue growth from ORLADEYO and a healthy cash position. See ...