People typically die from progressive supranuclear palsy within 7 to 10 years. There is currently no specialized treatment or effective screening for this neurodegenerative disease.
The dipeptidyl peptidase-1 (DPP1) inhibitor brensocatib became the first licensed therapy for bronchiectasis in the USA and Europe in 2025,1 based on the phase 3 ASPEN trial showing an approximate 20% ...
The drug leucovorin was touted by the Trump administration in September as a potential treatment for thousands of children with autism. Now, some parents and guardians of children with autism say they ...
Gepotidacin and nitrofurantoin demonstrate similar efficacy in reducing symptom severity and restoring daily function in women with uncomplicated UTI.
As IL-6 inhibition comes into practice for PMR, research advances on other therapeutic mechanisms of action, from IL-17 to ...
Keenova Therapeutics plc announced the first-time publication of a retrospective analysis using previously generated XIAFLEX® (collagenase clostridium histolyticum) data. The analysis evaluates ...
The Food and Drug Administration (FDA) announced a new proposal Monday for flexible drug approval pathway treatments addressing ultrarare diseases. The FDA unveiled draft guidance on a proposed ...
A nonprofit organization aiming to find a cure for a group of rare genetic skin disorders known as Epidermolysis Bullosa before the end of the decade is gaining ground on its goal by acting like a ...
A genetics‑guided small molecule called compound 6 gently turns down an overactive immune pathway (CARD9), offering a more precise, durable, and potentially safer treatment strategy for Crohn’s ...
Susannah Rosen was diagnosed with a KIF1A gene mutation at the age of 2½. Patients diagnosed with severe KIF1A gene mutations may live only into early childhood, while patients with mild symptoms can ...
This marks the first live procedure at AAHS, as well as a first for Keenova. Treatment takes place over two days of annual meeting—injection on Wednesday and finger extension on Friday. Audience of ...
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