rare-diseases
8 stories related to this topic, newest first.
StatTrump Administration Announces Projected Savings From Drug Pricing Agreements
The Trump administration is touting massive projected savings from still-secret drug pricing deals even as outside experts question how independently verifiable the figures are. STAT reported on the claims alongside fresh accusations of political interference at the FDA, which wi…
finance.yahoo.comFDA Shifts Remain Focus for Cell and Gene Therapy Industry at European Meeting
Cell and gene therapy developers gathered in Rome last week to discuss the sector's outlook in Europe. Conversations centered on recent FDA actions including a doubled rejection rate for such therapies since 2024 and the departure of the agency's top regulator for gene and cell t…
insidermonkey.comCall to Address Structural Barriers in Rare Disease Treatments Affecting Over 300 Million People
A report highlights rare diseases impacting more than 300 million individuals worldwide. It advocates for removing structural obstacles in treatment development. The piece proposes viewing certain therapies as molecular surgery rather than traditional drug treatments.
Substrate placeholder — needs reviewFDA Withdraws Approval for GSK's Leucovorin in Treating Cerebral Folate Deficiency
The U.S. Food and Drug Administration is withdrawing approval for GlaxoSmithKline's leucovorin as a treatment for cerebral folate deficiency, a rare brain disorder. GSK stated that the withdrawal was anticipated as part of the original approval plan. The disorder involves symptom…
Substrate placeholder — needs reviewRecent Advances in Gene Therapy for Rare Diseases Reported
Developments in gene therapy and targeted medications have shown progress in treating rare diseases such as spinal muscular atrophy and certain forms of blindness. These conditions affect an estimated 30 million people in the United States, with over 7,000 identified rare disease…
Substrate placeholder — needs reviewElevidys Gene Therapy Shows Positive Results in Duchenne Muscular Dystrophy Trial
Sarepta Therapeutics reported positive topline results from a phase 3 trial of its gene therapy Elevidys for Duchenne muscular dystrophy. The trial met its primary endpoint of improved motor function in patients aged 4 to 7. The Duchenne muscular dystrophy field has experienced s…
Substrate placeholder — needs reviewRecent Health News Highlights Breakthrough in Duchenne Treatment and Insulin Price Cap
A new therapy shows promise for Duchenne muscular dystrophy patients. Insulin prices are capped at $35 for many users. Additional evidence supports over-the-counter availability of abortion medications.
Substrate placeholder — needs reviewNeurocrine Biosciences Agrees to Acquire Soleno Therapeutics for $2.9 Billion
Neurocrine Biosciences has entered an agreement to purchase Soleno Therapeutics for $2.9 billion in cash. The acquisition aims to broaden Neurocrine's portfolio in treatments for rare genetic disorders. Soleno's lead product, DCCR, targets Prader-Willi syndrome, a rare condition…