NICE Approves Gene Therapy for Sickle Cell: A B...

By João L. Carapinha

February 4, 2025

Are we on the brink of a cure for sickle cell disease? NICE recently announced the approval of a one-off gene therapy sickle cell for severe sickle cell disease. Epidemiology of Sickle Cell Disease Sickle cell disease affects a signific...
Enhancing Rare Disease Decision-Making Through Integrated Data Linkage in Canada
Soaring Drug Prices in Europe: A Strain on Health Systems and Industry
NICE Collaborates with Menarini to Evaluate Elacestrant Breast Cancer Treatment
Understanding the Cost and Funding of Gene Therapies
The Power of Paediatric Biobanks: Advancing Child Health Research
Abu Dhabi Strategic Healthcare Partnerships: Global Collaborations
Genome-Wide Sequencing in Diagnosing Rare Diseases: Clinical and Economic Imp...
Rare Diseases: Modern Strategies for Enhanced Primary Care
Challenges and Opportunities in Educating the Public About Childhood Arthriti...
Access to Essential Medicines for Rheumatic Diseases in Children
Paediatric Rheumatology Care in Africa: Hope and Resilience
Juvenile Arthritis: Meeting the Benchmark with Health Technology