Chandigarh: Rare disease patients at PGIMER also fighting long wait for treatment
Despite eligible under central government’s National Policy for Rare Diseases, patients not receiving treatment at PGIMER, ...
ScienceAlert on MSN1d
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 ...
STAT1d
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 ...
Rare disease drug nitisinone makes human blood deadly to mosquitoes
A study in Science Translational Medicine found when patients take the drug nitisinone, their blood becomes deadly to ...
Dallas scientists granted $420K to test gene therapy for rare neurological disease
Childhood-onset striatonigral neurodegeneration robs children of the ability to walk and talk by age five. Researchers at ...
Soleno jumps as analysts say US nod for rare disease drug transforms treatment options
Shares of Soleno Therapeutics jumped 40% in morning trading on Thursday, after the company's drug for a rare metabolic condition became the first such treatment to receive U.S. regulatory nod.
Rolling Out1d
Promising drug cuts Alzheimer’s risk by half in rare cases
Research shows experimental anti-amyloid drug reduced cognitive symptom risk by 50% in people with rare genetic mutations ...
An ‘Impossible’ Disease Outbreak in the Alps
In one tiny town, more than a dozen people were diagnosed with the rare neurodegenerative disease ALS. Why? I n March 2009, ...
Zacks Investment Research on MSN19h
IONS Out-Licenses ex-U.S. Rights for Rare Disease Drug Tryngolza to Sobi
Ionis Pharmaceuticals IONS signed a license agreement with Sweden-based Sobi. Per the terms, Sobi will acquire exclusive ...
FDA Approves Expanded Use Of Novartis Rare Disease Drug As Only Treatment For Rare Type Of Kidney Disease
Novartis' Fabhalta receives FDA approval for C3 glomerulopathy, expanding its kidney disease portfolio as global regulatory ...
Cherokee Phoenix13d
Cherokee Nation Rare Disease Task Force outlines recommendations tribe can take to assist those with rare diseases
The Cherokee Nation Health Services Rare Disease Task Force has released a report outlining progress made over the past year ...
STV News13h
Hebridean Blue Cheese recalled over fears it may cause rare kidney disease
Isle of Mull Cheese has issued an urgent 'do not eat' warning on the product because of contamination with pathogenic E.coli.