Lafora disease is a rare, fatal genetic disorder affecting the brain, with limited treatment options and severe symptoms.

Epidermolysis Bullosa, also colloquially known as butterfly skin disease, a rare genetic condition that makes your skin very ...

The administration has approved 120 billion rials (some 144,000 dollars) for treating rare and hard-to-treat diseases in the ...

The landmark achievement in diabetes research in the country could potentially transform how certain rare forms of the ...

Scientists have made an important new discovery in medical research, identifying a rare disease called Lyn kinase-associated ...

The FDA has approved Abeona Therapeutics’ prademagene zamikeracel (Zevaskyn) gene therapy for recessive dystrophic epidermolysis bullosa (DEB), a rare genetic skin disease. Prademagene ...

Three American siblings and six other children born with a rare, life-threatening disease are returning to normal life thanks ...

"It's a momentous breakthrough for families facing this devastating disease," said Dr. Claire Booth, leader of the London ...

The FDA has approved Zevaskyn (prademagene zamikeracel) for the treatment of recessive dystrophic epidermolysis bullosa, a rare genetic skin disorder.

Discover a study in which the analysis demonstrated significant reductions in HAE attacks among adolescent patients.

Sindrom cri du chat adalah salah satu sindrom genetik langka yang bisa terjadi pada bayi. Ini bisa ditandai dengan suara ...

Deep brain stimulation at the MHH saves two sisters from a wheelchair Nele (22) and Jette (18) R. are cheerful young women who go their own way with confidence. And they are sisters. But apart from, ...