Genetic Disorders

Sickle Cell Disease Rare Diseases Trisomy 18 Gene Therapy Angelman Syndrome Neurofibromatosis Cancer Cancer Research Mitochondrial Diseases Deafness

Precision RNA Therapy Reverses Progeria in Preclinical Models

The reversible RNA scissors technique eliminates mutant progerin transcripts to restore tissue function in progeria models

Top Chef Winner Hosea Rosenberg Updates on Daughter Sophia’s Rare Bone Disorder

UK Probes Safety of Three-Parent IVF After Eight Births and Mitochondrial Reversions

FDA Grants Orphan Designation to AI-Identified Vorinostat for Rett Syndrome Trial

Scientists Identify Hemifusome, a New Organelle in Cellular Cargo Sorting

India Approaches 6 Crore Screenings Under National Sickle Cell Mission

Vigo Hospital Administers Spain’s First Public Gene Therapy for Hemophilia B

Nicotinamide Riboside Improves Cardiovascular, Skin and Kidney Health in Werner Syndrome

Personalized CRISPR Therapy Sends Baby KJ Home from CHOP

Manchester mother struggles with social isolation from rare 'fish odour' disorder

FDA Halts Rocket’s RP-A501 Gene Therapy for Danon Disease After Patient’s Death

Jennifer Saro Confirms Plans for a Larger Family and Relocation to Dortmund

Landmark Study Maps Rapidly Mutating DNA Regions Across Four Generations

Researchers Release AI-Driven Tool for Custom CRISPR-Cas9 Enzyme Design

Colin Farrell Faces Challenges in Securing Long-Term Care for Son with Angelman Syndrome

Breakthrough Liver Organoids Advance Regenerative Medicine and Drug Testing

Influencer Hannah Campbell Mourns Death of 10-Month-Old Daughter From Rare Genetic Disorder

Nitisinone Identified as Promising Mosquito-Killing Tool in Malaria Fight

Prince Frederik of Luxembourg Remembered for Advocacy Amid Rare Disease Battle

Prince Frederik of Nassau Dies at 22 After Battle with Rare Genetic Disease