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, ...
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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 ...
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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.
Drug treatment extended for Langford girl with rare fatal condition
The family of nine-year-old Charleigh Pollock will continue to be covered for the drug, which costs $1 million a year, until ...
Delhi HC denies interim injunction on Roche rare disease drug, opening the door for generic versions
Patients with Spinal Muscular Atrophy (SMA) are hopeful of getting access to generic versions of Roche’s patented drug ...
After a Rare Diagnosis, This Man Found Strength in Fitness. Now, His Story Is Inspiring Others
A neuromuscular disease put Anthony Monaco in a wheelchair. But it hasn't stopped him from competing in CrossFit.
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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, ...
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 ...