Rare Disease News and Research

RSS
Mayo Clinic and nference launch startup to develop drugs for rare diseases

Mayo Clinic and nference launch startup to develop drugs for rare diseases

FDA to expedite orphan drug processing queues

FDA to expedite orphan drug processing queues

Scientists discover link between sugar metabolism and peroxisomal biogenesis disorders

Scientists discover link between sugar metabolism and peroxisomal biogenesis disorders

Blocking newly discovered cellular pathway could help control tumor growth

Blocking newly discovered cellular pathway could help control tumor growth

When an insurer balks and treatment stops

When an insurer balks and treatment stops

Statin medications can cause necrotizing myopathy in some patients

Statin medications can cause necrotizing myopathy in some patients

Insilico Medicine launches personalized drug discovery platform to develop new treatments for ALS

Insilico Medicine launches personalized drug discovery platform to develop new treatments for ALS

Collaborative effort to develop rare disease patient registries

Collaborative effort to develop rare disease patient registries

Protein that helps regulate childhood growth could become possible target for anticancer therapies

Protein that helps regulate childhood growth could become possible target for anticancer therapies

FDA expands approval for cystic fibrosis drug to treat additional rare gene mutations

FDA expands approval for cystic fibrosis drug to treat additional rare gene mutations

Spanish scientists develop promising gene therapy method to treat Wilson's disease

Spanish scientists develop promising gene therapy method to treat Wilson's disease

Boosting clinical trial research in London

Boosting clinical trial research in London

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

New findings pinpoint key feature of rare childhood disease that could help fast-track diagnosis

New findings pinpoint key feature of rare childhood disease that could help fast-track diagnosis

Study shows how influenza A hijacks human cellular machinery to replicate

Study shows how influenza A hijacks human cellular machinery to replicate

FDA approves new treatment for people with ALS

FDA approves new treatment for people with ALS

International consensus addresses research bottlenecks to diagnose rare genetic diseases

International consensus addresses research bottlenecks to diagnose rare genetic diseases

Rat study reveals new clues to devastating neurological disease

Rat study reveals new clues to devastating neurological disease

Targeted treatment slows progression of two degenerative brain diseases in mice

Targeted treatment slows progression of two degenerative brain diseases in mice

Scientists discover potential trigger for MDS in children with rare genetic disorder

Scientists discover potential trigger for MDS in children with rare genetic disorder