Fairfax Co. teen turns rare disease diagnosis into youth-led support, advocacy
Khartik Uppalapati, 16, of Fairfax County, Virginia, has co-founded an advocacy group aimed at helping young people, like ...
The National Institute for Health and Care Excellence14h
HST routing criteria: encouraging innovation in the treatment of ultra-rare diseases
Our chief medical officer, Professor Jonathan Benger, explains the crucial role the HST programme plays in encouraging ...
ET HealthWorld4d
Around 1,000 patients with rare diseases registered with Centres of Excellence, LS told
"Health being a state subject, the primary responsibility of providing healthcare services is that of state governments.
MPR5h
Pegcetacoplan Gets Priority Review for Two Rare Kidney Diseases
The supplemental New Drug Application for the C3G and IC-MPGN indications is supported by data from the phase 3 VALIANT study.
Once-Rare Fungal Diseases Are Killing Millions in an Unprepared World
When most people think of dangerous infections, they picture bacteria or viruses. But for infectious disease specialists like ...
Scientists granted $420,000 to test gene therapy for rare neurological disease
Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...
Live Science on MSN1d
In 'extremely rare' case, Michigan resident dies from rabies after receiving transplanted kidney carrying the virus
A Michigan resident has died from rabies following a kidney transplant at a hospital in Ohio. Investigations show that the ...
ScienceAlert on MSN6d
Drug For Rare Disease Turns Human Blood Into Mosquito Poison
Scientists have a radical new plan for controlling mosquito numbers and fighting malaria: lacing human blood with a drug that ...
Retiring country music icon has rare disease. Here’s what we know
He took the stage and was great last week at the Grand Ole Opry’s 100th anniversary show, and now he is down to just two ...
My Son’s Disease Means I Know the Price of Trump’s Idiots’ ‘Mistakes’
My son is 8 and doctors are saving his life but worries we can go extinct from stupidity. He’s not wrong. News Editor Allison ...
Science Daily6d
Rare disease drug nitisinone makes human blood deadly to mosquitoes
Nitisinone was shown to last longer than ivermectin in the human bloodstream, and was able to kill not only mosquitoes of all ages -- including the older ones that are most likely to transmit malaria ...
STAT6d
Soleno Therapeutics’ ‘life-changing’ rare-disease drug approved
The FDA Wednesday approved the first drug to treat people with Prader-Willi syndrome, a rare genetic disease that causes an ...