A 44-year-old man presented with a 5-year history of heat intolerance and burning pain in his hands and feet. He had periumbilical angiokeratomas and nonnephrotic proteinuria. A kidney biopsy was ...

Gene therapy company uniQure disclosed that the Food and Drug Administration granted orphan drug designation to AMT-191, a potential treatment for Fabry disease. Fabry disease is a genetic ...

LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing ...

New Delhi: The Central government is set to launch a scheme to develop 12 indigenous drugs for eight rare diseases, health ...

The FDA has cleared an IND for a phase I/II trial of LA-GLA (GC-1134A, HM-15421), an innovative enzyme replacement therapy for Fabry disease being co-developed by GC Biopharma Corp. and Hanmi ...

Sep. 25, 2024 — Abdominal body fat could hint at cardiovascular disease risk even when other health factors are not considered, a new study ... Researchers Discover New Target for Treating Heart ...

Moreover, the company has a genetic disease program focused on Fabry disease, which is in the preclinical stages. RLAY also has programs for vascular malformations in the preclinical and early ...

Parkinson’s disease is a devastating condition that progressively causes trouble with movement, mobility, and speech. But while Parkinson’s disease has long been considered a disease of the ...

Patients with chest pain/ischemic cardiac disease and normal coronary arteriography are thought to have a benign prognosis despite diminished quality of life. Many patients with hypertension fall ...

Oct. 3, 2024 — An international team of researchers have made a promising breakthrough in the development of drugs to treat Alzheimer's Disease. For the first time, scientists have developed a ...