tldrdaily.news logo
Tag

Rare Disease

All articles tagged with #rare disease

FDA reversal sinks rare-disease cell therapy after favorable reviews
healthcare3 hours ago

FDA reversal sinks rare-disease cell therapy after favorable reviews

An experimental cell therapy for a rare post-transplant blood cancer, developed by Atara Biotherapeutics and Pierre Fabre, was on track for FDA approval after internal reviewers recommended clearance, but the agency unexpectedly rejected it last month, citing deficient clinical data. Anonymous former agency sources say the decision may reflect leadership changes, marking a sharp reversal that disrupts a treatment expected to help roughly 500 patients in the US each year with a grim prognosis.

via statnews.com|
#biotech#drug-approval#fda
Two-Year-Old’s Kawasaki Disease Battle Ends Weeks Before Birthday
health1 month ago

Two-Year-Old’s Kawasaki Disease Battle Ends Weeks Before Birthday

A 2-year-old boy, Hudson Hughie Martin, died after a Kawasaki disease diagnosis, a rare inflammatory illness that can resemble flu-like symptoms and affect the heart; diagnosed at 7 months, he underwent two rounds of treatment but developed a heart aneurysm and died following a collapse at home on Jan. 8. His parents say there is no definitive test for Kawasaki disease and urge awareness of its signs to ensure timely care, as a GoFundMe fundraiser was set up to support the family.

via Yahoo News Canada|
#gofundme#health#hudson-hughie-martin
Repurposed DFMO Could Bring Hope for Bachmann-Bupp Syndrome
health1 month ago

Repurposed DFMO Could Bring Hope for Bachmann-Bupp Syndrome

Decades-old DFMO (eflornithine), an inhibitor of the ODC1 enzyme, is being explored as a therapy for Bachmann-Bupp syndrome, an ultra-rare neurodevelopmental disorder. Early use in a handful of patients—some via FDA-approved single-patient protocols—has shown symptom improvements. A collaboration among Corewell Health, Michigan State University, and Every Cure aims to build preclinical data and streamline regulatory paths toward a formal clinical trial, addressing awareness, study design, and endpoints. With about 20 cases identified worldwide, the group plans a preclinical study next year to bolster evidence and help bring potential DFMO therapy to more patients.

via SciTechDaily|
#bachmann-bupp-syndrome#dfmo#drug-repurposing
Healthy Baby's Sudden Decline Due to Rare, Unknown Disorder
health5 months ago

Healthy Baby's Sudden Decline Due to Rare, Unknown Disorder

A young boy named Jack, born prematurely and initially healthy, was diagnosed with a rare, terminal genetic disorder called INAD, which leads to progressive loss of motor and cognitive functions. Despite no current cure, a promising gene therapy is in development, and a community-funded effort is underway to fund clinical trials that could extend Jack's life and improve his quality of life. The family remains hopeful and focused on making the most of their time together.

via Yahoo|
#child-health#clinical-trial#gene-therapy