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 ...

She said they kept telling her, “Girls don’t get Duchenne.” Of the hundreds of genetic diseases linked to the X chromosome, Duchenne is among the more common, along with certain forms of hemophilia.

Interest in lysosomal storage diseases (LSDs) has increased significantly, because several new therapeutics received FDA approval in 2003. Though long overlooked by most major pharmaceutical ...

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

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 ...

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

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 ...

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 ...

“This important designation highlights the need for new gene therapies like AMT-191 for patients with Fabry disease with the potential of delivering meaningful benefit given the suboptimal ...

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 ...