Researchers decry ‘disastrously bad idea’ as NIH slashes payments for research infrastructure
The US National Institutes of Health is lowering the maximum “indirect cost rate” that research institutions can charge the ...
MedPage Today22h
Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
Medscape1d
Could Gene Therapy for RA Soon Be Ready to ‘Take Off’?
Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...
Daily Nation on MSN2d
Groundbreaking treatment for sickle cell disease approved for use
Treatment for sickle cell disease, the most common inherited blood disorder in Kenya, has been approved for use in England for older children and adults with a severe form of the disease.The treatment ...
precisionmedicineonline8d
NICE Recommends Vertex, CRISPR Therapeutics' Casgevy for Sickle Cell Under Managed Access Deal
The gene-editing treatment will be available to certain patients with sickle cell disease in England as Vertex commits to collecting additional data.
STAT8d
Pharmalittle: We’re reading about FDA OK for Vertex non-opioid pain drug, vaccine advisory committee, and more
Vertex Pharmaceuticals won approval from the U.S. Food and Drug Administration for a non-opioid pain drug, clearing the way ...
Channel 4 News8d
Sickle cell disease: new treatment raises hopes of cure for sufferers
The Exa-cel gene therapy treatment is at the forefront of medical technology. It takes the person’s DNA and “edits” out the faulty sickle gene. It’s expensive, too. According to the manufacturer, ...
Pharmaceutical Technology8d
NHS England to offer Casgevy for sickle cell disease patients
The UK’s Medicines and Healthcare Regulatory Agency (MHRA) approved Casgevy for sickle cell disease in 2023, followed by ...
National Institute for Health and Care Excellence8d
Groundbreaking one-off gene therapy approved for severe sickle cell disease
People in England to benefit from groundbreaking one-off gene therapy for severe sickle cell disease approved by NICE ...
Sickle cell disease ‘cure’ to be offered to NHS patients
Around 1,700 people could be eligible for the one-time treatment, which is seen as a less risky alternative to a donor stem ...
health.economictimes.indiatimes8d
UK health system to offer cutting-edge gene therapy for sickle cell disease
The therapy involves taking stem cells out of a patient's bone marrow and editing a gene in the cells in a laboratory and ...
Science Media Centre8d
expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease
January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...