As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...

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.

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 ...

It is hoped this will enrich the FindZebra.com search engine to enhance earlier and more accurate diagnosis of Gaucher and Fabry diseases by developing specialised search tools. “That’s a nice ...

Feb. 27, 2025 — A new study suggests a nasal spray developed to target neuroinflammation could one day be an effective treatment for traumatic brain injury (TBI). By studying the effects of the ...

Alzheimer's disease is a progressive neurodegenerative disease that impairs memory and cognitive judgment and is often accompanied by mood swings, disorientation and eventually delirium.

Sex differences in a mouse model of synucleinopathy reveals female resilience to disease progression despite early brain pathology, contrasting with accelerated disease in male mice after alpha ...

2-Year U.S. Treasury Note Continuous Contract $103.488 0.004 0.00% 5-Year U.S. Treasury Note Continuous Contract $107.961 0.023 0.02% 10-Year U.S. Treasury Note Continuous Contract $111.172 0.078 ...

Lei Jun set out to build the ‘Apple of China.’ Xiaomi’s car business is now outpacing Tesla and Rivian. A lack of trust and the whack-a-mole nature of stamping out the chemicals used to make ...

Division of Cardiology, Department of Medicine, Emory University School of Medicine, Atlanta, GA (B.R.R., N.W.D.). Division of Cardiology, Department of Medicine ...