Acsh.org1d
Rare Diseases Are Neglected. The FDA Needs A Dedicated 'Center of Excellence for Rare Diseases'.
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...
Gene variant could protect against ultra-rare seizure disorder, fruit fly research finds
One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...
News Medical on MSN1d
AI approach reveals possible fusion of rare diseases in COVID-19 origins In A Landmark Dis...
Despite extensive research, the origins of COVID-19 remain elusive. In a new study published in the KeAi journal Advances in Biomarker Sciences and Technology (ABST), an AI-driven approach was adopted ...
‘Noah,' a Documentary About a Woman With Seven Rare and Incurable Diseases, Acquired by Jambika Docs (EXCLUSIVE)
Barcelona-based sales agency Jambika Docs has acquired the documentary feature "Noah" ahead of the European Film Market in ...
GlobalData on MSN7h
Travere takes rare disease kidney drug to FDA despite failed trial
Travere’s stock price climbed by 14% following the announcement that it will seek an FDA label expansion for Filspari.
STAT6d
What happens when a rare disease drug seems to work, but the health care system doesn’t?
Cristina Brennan can hear the clock ticking. At the end of the year, her 12-year-old son Tristan will no longer have free ...
EurekAlert!2d
Uncovering two rare infectious diseases which might be responsible for COVID-19
In a landmark discovery, researchers applied a novel AI model called the max-logistic intelligence classifier to analyze ...
FiercePharma7h
Amid takeover talk, SpringWorks scores FDA nod for rare genetic disease treatment Gomekli
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...
Foodborne botulism is a rare, life-threatening condition. These are the signs to look for.
Canned foods are often the culprit of this rare, but potentially deadly, condition. The good news is: It's treatable if ...
KTBS20h
Texarkana boy battling rare disease prepares for life-changing transplant
TEXARKANA, Texas - A Texarkana, Texas boy battling a rare genetic disease is preparing to head to Arkansas Children's ...
India Today13d
Rare disease patients in crisis as delays stall national policy implementation
Patient advocacy groups warned that delays in implementing India's rare disease policy have left many, including children, in ...
Yen.com.gh3d
Mfantsipim Old Boy Who Has Been Standing For 5 Years Opens Up About Rare Disease
A first-year University of Cape Coast student and Mfantsipim old boy, Enock Eshun has been battling a rare disease that has ...