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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

Prevention, not cure, may be key to stopping Alzheimer’s disease

Prevention, not cure, may be key to stopping Alzheimer’s disease

Researchers achieve new step towards treatment of myotubular myopathy using gene therapy

Researchers achieve new step towards treatment of myotubular myopathy using gene therapy

Liver shows positive response to leptin therapy in patients with partial lipodystrophy, study finds

Liver shows positive response to leptin therapy in patients with partial lipodystrophy, study finds

Discovery of new genes linked to  Erdheim-Chester disease provides hope for improving diagnoses

Discovery of new genes linked to  Erdheim-Chester disease provides hope for improving diagnoses

NHGRI researchers use facial recognition software to diagnose rare genetic disease

NHGRI researchers use facial recognition software to diagnose rare genetic disease

OTUD6B gene mutations linked to physical and intellectual deficits

OTUD6B gene mutations linked to physical and intellectual deficits

Genome sequencing approach opens door to therapy options for rare, neglected cancers

Genome sequencing approach opens door to therapy options for rare, neglected cancers

Researchers use animal model to study brain abnormality in human TD patients

Researchers use animal model to study brain abnormality in human TD patients