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 ...
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 ...
Mother Jones6d
Project 2025 Is Gutting Medical Funding That Helped Russell Vought’s Own Kid
Its architect's daughter has cystic fibrosis—and benefits from a "miracle drug" backed by an agency he's attacking.
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 ...
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 ...
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.
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.