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Rare Disease

All articles tagged with #rare disease

FDA Grants Accelerated Approval for KRESLADI to Treat Pediatric LAD-I
healthcare14 days ago

FDA Grants Accelerated Approval for KRESLADI to Treat Pediatric LAD-I

Rocket Pharmaceuticals announced that the FDA granted accelerated approval for KRESLADI (marnetegragene autotemcel), an autologous hematopoietic stem cell–based gene therapy for pediatric patients with severe LAD-I due to ITGB2 variants who lack an HLA-matched donor. Approval is based on increased neutrophil CD18/CD11a expression and will rely on longer-term follow-up data and a post‑marketing registry to confirm benefit. The FDA also awarded Rocket a Rare Pediatric Disease Priority Review Voucher, which the company plans to monetize. LAD-I is ultra-rare and causes recurrent life-threatening infections; safety concerns include infections during conditioning, veno-occlusive disease, engraftment failure, potential insertional oncogenesis, hypersensitivity, anti-retroviral interactions, and possible HIV test false positives, necessitating long-term monitoring. A conference call is planned for March 27, 2026.

via Business Wire|
#fda-approval#gene-therapy#healthcare
Tiny bite, big danger: Arizona woman fights botulism from fermented swordfish
health24 days ago

Tiny bite, big danger: Arizona woman fights botulism from fermented swordfish

A 24-year-old Arizonan developed botulism after sampling a small piece of fermented swordfish, leading to paralysis and respiratory failure. She required antitoxin and a respirator, and two friends who tried the swordfish also fell ill. She is recovering and could be discharged soon, planning to avoid fish for now; experts note food botulism remains rare in the U.S., with only about two dozen cases annually.

via Yahoo|
#arizona#botulism#health
Rare botulism from homemade swordfish leaves Phoenix woman on ventilator
health27 days ago

Rare botulism from homemade swordfish leaves Phoenix woman on ventilator

A 24-year-old Phoenix wedding planner developed life-threatening botulism after she and friends ate a homemade fermented swordfish. She required hospitalization, a ventilator, and an antitoxin; two friends also contracted botulism. The illness is extremely rare in the U.S., with about two dozen cases annually, and her recovery is expected to be lengthy.

via KUSA.com|
#botulism#fermentation#foodborne-illness
Arizona woman battles rare botulism after shared meal of fermented swordfish
health28 days ago

Arizona woman battles rare botulism after shared meal of fermented swordfish

A 24-year-old Arizona wedding planner developed life-threatening botulism after sharing a friend's homemade fermented swordfish; she required ventilation and an antitoxin at Barrow Neurological Institute and is slowly recovering, while five friends who ate the dish include two who also contracted botulism. A fundraiser has been started to cover medical costs, and doctors note that food botulism is extremely rare, with about two dozen U.S. cases annually.

via KING5.com|
#arizona#botulism#fermented-swordfish
EB survivor pushes Medicare study to cover bandages
health28 days ago

EB survivor pushes Medicare study to cover bandages

Shane DiGiovanna, born with epidermolysis bullosa, a rare condition that makes skin as fragile as a butterfly’s wing, helped spur the Shane DiGiovanna Act to require a Department of Health and Human Services study on Medicare coverage of bandages for EB, addressing massive out-of-pocket costs and state coverage gaps; the bill, introduced by Rep. Greg Landsman, seeks bipartisan support and would pave the way for potential coverage mandates if the study finds long-term savings, all while DiGiovanna remains in hospice care with terminal cancer.

via WKRC|
#bandage-costs#epidermolysis-bullosa#health
FDA keeps leucovorin for a rare condition, not autism
health-policy1 month ago

FDA keeps leucovorin for a rare condition, not autism

The FDA said leucovorin will be approved only for cerebral folate deficiency (CFD-FOLR1) and will not be expanded to autism, despite the Trump administration’s push. Regulators cited limited evidence for autism treatment and noted a retraction of a key study, narrowing the review to the rare CFD-FOLR1 condition and highlighting the gap between political claims and scientific proof for autism therapies.

via Ars Technica|
#autism#cfd-folr1#fda
FDA approves leucovorin for cerebral folate deficiency in rare patients
health1 month ago

FDA approves leucovorin for cerebral folate deficiency in rare patients

The FDA approved leucovorin (folinic acid) as the first treatment for cerebral folate deficiency—a rare genetic disorder that prevents folate from reaching the brain—in select adults and children. The decision was based on a systematic review of published case data rather than a randomized trial and did not establish broad efficacy for autism, though future autism studies remain possible. The approval covers generic leucovorin and GSK’s Wellcovorin, and regulators urged manufacturers to boost production to meet rising demand; GSK has no plans to relaunch the product.

via CNBC|
#autism#cerebral-folate-deficiency#fda-approval
Biotech investors wary as FDA shifts stance on rare-disease drugs
business1 month ago

Biotech investors wary as FDA shifts stance on rare-disease drugs

Over the past year the FDA has denied or discouraged at least eight drug applications, including UniQure’s Huntington’s gene therapy and Regenxbio’s Hunter syndrome, and even reversed course on Moderna’s flu vaccine review, prompting investors to doubt whether the agency’s rare-disease flexibility will endure and to worry about the fate of other pipeline drugs; upcoming decisions (e.g., Denali Therapeutics) will test whether current standards remain consistent amid broader regulatory uncertainty.

via CNBC|
#biotech#business#drug-approval
Nihal's UV Shield: living with xeroderma pigmentosum in France
health1 month ago

Nihal's UV Shield: living with xeroderma pigmentosum in France

Nihal was diagnosed with xeroderma pigmentosum at two and a half and must live under near-total UV protection—helmet, long coat, gloves—because sun and some artificial lights can cause severe damage and raise skin cancer risk by about 4,000 times. With support from the Enfants de la Lune association, she attends UV-filtered school spaces, pursues indoor sports, and leads a life that is almost normal: she has become a freelancer in Saint-Étienne, married in 2025, and dreams of a family, while staying connected with other “Children of the Moon” through annual gatherings that help cope with daily challenges and stigma.

via The Guardian|
#france#health#rare-disease
FDA reversal sinks rare-disease cell therapy after favorable reviews
healthcare1 month 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
health2 months 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