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

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 ...

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 ...

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

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 ...

Jan. 16, 2025 — Two new studies have identified a possible way to block the progression of several forms of blood cancer using a drug already in clinical trials ... New Innovative Local ...

The global Gene Therapy market, valued at US$7.21 billion in 2023, is forecasted to grow at a robust CAGR of 19.4%, reaching US$8.85 billion in 2024 and an impressive US$36.55 billion by 2032. Market ...