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, ...
KSN-TV on MSN15h
Kansas nonprofit gains new partnership to help families battling rare diseases
Coming Together for a Cure partnered with Cariloop to give families free access to its caregiver support platform.
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 ...
LJ Smith dead: The Vampire Diaries author who inspired TV show dies after battle with rare disease
Author LJ Smith, who was behind the Vampire Diaries series that inspired the hit TV show of the same name, has died at the ...
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 ...
Country music icon retiring with rare disease. Here’s what to 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 ...
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, ...
BioPharma Dive7d
Biotech Community Curetopia Raises $1.77M to Advance Rare Disease Cures with Blockchain
In a groundbreaking achievement, Curetopia, the first Biotech Decentralized Autonomous Organization (DAO) built natively on ...
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 ...
LiveLaw2d
Availability Of Drug For Rare Diseases At Economical Prices Material Factor To Be Considered Before Restraining Via Interim Injunction: Delhi HC
The Delhi High Court has observed that availability of a drug for treatment of rare diseases at economical and competitive ...
US FDA expands approval for Alnylam's drug to treat rare heart disease
U.S. Food and Drug Administration approved Alnylam's drug to treat a rare and deadly heart disease on Thursday, allowing the ...
Euractiv9d
Bulgarian rare disease patients urge government to fund compassionate drug use
Insufficient screening, very low age limits, and problematic diagnosis continue to disproportionately affect patients with ...