Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ...

Researchers created a 1-atom-thick carbon membrane that sharpens proton beams, improving cancer therapy and enabling cleaner, scalable tech across multiple fields. (Nanowerk News) Researchers from the ...

An injectable cure for inherited heart muscle conditions that can kill young people in the prime of their lives could be available within a few years, after an international team of researchers were ...

TOKYO--(BUSINESS WIRE)--B dot Medical Inc. has succeeded in high-speed scanning irradiation for an ultra-compact proton therapy system under development. That confirmed that the system under ...

Michael Pirovolakis was diagnosed with spastic paraplegia type 50 (SPG50) when he was 18 months old. SPG50 is an “ultra-rare” progressive neurodegenerative disorder that causes developmental delays, ...

Bone marrow aspirates and peripheral blood samples (leukemic cells, 39 samples) and skin biopsies (normal tissue for germline genomic information) from 12 AML patients and one high-risk (according to ...

Numerous cell and gene therapy solutions have exhibited promise as a source of potential treatment for an extensive range of chronic disorders. However, these innovative therapies typically introduce ...

PTC Therapeutics’ Upstaza, a gene therapy for an ultra-rare genetic disorder and the first to be directly infused in the brain, is one step closer to reaching patients in the U.K. The National ...

Collaboration Aims to Establish Sustainable Access to a Newly Approved Therapy Targeting a Rare Immunodeficiency, Filling Urgent Gap Left by For-Profit Efforts CAMBRIDGE, Mass. and ROME, Dec. 15, 2025 ...

SAN DIEGO — Doctors were just guessing a decade ago when they gave Alison Cairnes' husband a new drug they hoped would shrink his lung tumors. Now she takes it too, but the choice was no guesswork.

During his 2013 California nursing-home stay, Jack Furumura became severely dehydrated and shed more than 5 pounds, partly because staff didn’t follow written plans for his nutrition or the facility’s ...