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 ...
bangaloremirror.indiatimes2d
Too little, too late: Rare disorder patients suffer
In India, 32 patients, diagnosed with this condition by the Centres of Excellence (CoEs), have registered themselves with the ...
His daughter has an ultra-rare genetic disorder. His push to treat it just hit a major milestone
A clinical trial by Shionogi is a milestone in the search to treat Jordan's Syndrome, a rare genetic disorder named after a ...
technologynetworks8d
Tackling Rare Diseases With RNA Therapeutics
To learn about the progress being made in the RNA therapeutics space, we spoke to CTO and Co-Founder of La Jolla Labs, Jeff ...
Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3
Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative ...
Rare disease patients and advocacy groups appeal to PM Modi for sustainable funding for treatment
Families of rare disease patients appeal to PM Modi and Health Minister for increased treatment funding support.
Carlsbad foundation offers hope for 3-year-old with rare terminal genetic disease
The nonprofit foundation, n-Lorem developed personalized medicine for a child with rare leukodystrophy.
Business Wire7d
New Interactive Storybook Aims to Educate Children with Rare Kidney Disease into a World of Adventure and Understanding
Estimated to affect 0.4 - 0.5 people per million, 1 aHUS is a genetic, ultra-rare disease, which can progressively damage vital organs, such as the kidneys, and can affect both children and adults.
pharmaphorum7d
Storybook can help kids understand rare kidney disease
aHUS is an ultra-rare disease caused by abnormalities in the complement system that is estimated to affect only around one person in every two million. It leads to progressive damage to the ...