Amicus Therapeutics’ first oral drug for the rare condition Fabry disease has been approved by the FDA, giving patients an alternative to conventional injected enzyme replacement therapy.

The predominant inheritance pattern is autosomal recessive except for Anderson-Fabry disease, glycogen storage disease (GSD) type IIb (Danon disease) and mucopolysaccharidosis (MPS) type II (Hunter ...

ukmuenster.de Background Patients with Fabry disease (FD) on reduced dose of agalsidase-beta or after switch to agalsidase-alfa show a decline in chronic kidney disease epidemiology ...

June 10, 2024 — Scientists have grown 'mini-guts' in the lab to help understand Crohn's disease, showing that 'switches' that modify DNA in gut cells play an ... Major Cause of Inflammatory ...

Jan. 2, 2025 — Patients diagnosed with late-stage or metastatic breast cancer have a statistically significant increased risk of pre-diagnosis cardiovascular disease (CVD) compared to those with ...

The disease has infected mainly women and children, resulting in 143 deaths. A deadly flu-like disease that has claimed the lives of more than 143 people in the Democratic Republic of the Congo ...

A previously unknown disease making the rounds in the Democratic Republic of Congo (DRC) is a severe form of malaria, the country’s health ministry has announced. Health authorities on Tuesday ...