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Rare Diseases

Treatment Options Sarcomas Brain Tumors AnWj-negative Individuals Hereditary Hemorrhagic Telangiectasia Neurological Disorders Niemann-Pick Disease Arthrogryposis Diagnosis Genetic Conditions Neurodevelopmental Disorders Anemia Epidemiology Creatine Transporter Deficiency Research Foundations Support Organizations Autoimmune Disorders Pediatric Conditions Pediatric Rare Diseases Patient Care Cancer Treatments

Five-Year-Old Rosie Hassall Battles Ultra-Rare Brain Tumour with 9–12 Month Prognosis

Diagnosed with a new entity of paediatric bithalamic glioma, Rosie continues chemotherapy as her family raises funds and awareness for research.

Merck Announces €3 Billion Acquisition of Springworks Therapeutics

HiFi Sequencing Reveals Tenfold Increase in Genomic Imprinting Events

Madrid Detects First SCID Case Through Neonatal Screening Program

NHS Launches Groundbreaking Genetic Testing for Newborns

AI Tool Identifies Drug Candidates for Over 17,000 Rare Diseases

FDA Approves First Treatment for Niemann-Pick Disease Type C

Scientists Discover New MAL Blood Group, Solving 50-Year-Old Mystery

FDA Approves Adaptimmune's Groundbreaking T-Cell Therapy for Rare Cancer