A new federal grant is allowing Moffitt Cancer Center to study a rare and aggressive disease that affects cancer patients, ...

The US Food and Drug Administration (FDA) has selected Abeona Therapeutics’ ABO-503 gene therapy for X-linked retinoschisis ...

In the journal Childhood in the Past, Ph.D. candidate Duru Yağmur Başaran published the results of an analysis of an over 900 ...

The centerpiece of the collaboration is the gene editor ABO-101, being developed for primary hyperoxaluria type 1, a rare ...

Working with agency partner DCX Growth Accelerator, GeneDX takes direct aim at the issue of underdiagnosis of pediatric rare diseases.

UCB's experimental medicine for rare disease thymidine kinase 2 deficiency (TK2d) has been shown to reduce the risk of death ...

Rare diseases are complicated and communicating reliable, accessible health information around them can be even more complicated. Some conditions like RPI deficiency or Aicardi syndrome affect less ...

Cade was diagnosed with a rare, neurodegenerative disease when he was 2½. Now 4, Cade has been receiving treatment for the ...

Since the 1960s, Texas has screened newborns for a variety of rare diseases — like sickle cell anemia and cystic fibrosis.

In a closely watched case, Roche suffered a setback as a court said it would permit a generic version of its rare disease ...

For Ms Miria Mukiibi Kibirige, that struggle is a daily reality. She cares for two children living with epidermolysis bullosa (EB), a rare genetic skin disorder causing painful, life-threatening ...