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May 03, 2026
C-peptide is described as a key biomarker for beta cell function in type 1 diabetes and a potential surrogate endpoint for drug approval. Current trial endpoints remain difficult for disease-modifying therapies.
Mar 25, 2026
Researchers have identified a new genetic disease characterized by premature aging and cognitive deficits, tracing it to a mutation in the IVNS1ABP gene. Using cellular reprogramming, they discovered the mutation causes cellular senescence and DNA damage during cell division through altered actin dynamics. The findings highlight the potential of patient-derived stem cell models to study rare diseases and identify potential treatment approaches.
Mar 17, 2026
Recent research identifies novel genetic causes for rare movement and neurodegenerative disorders, including CD99L2 variants in spastic ataxia, PPP2R5C as an Alzheimer's biomarker, and toxic polyglycine proteins in GGC repeat expansion diseases.
Mar 05, 2026
The FDA granted Breakthrough Therapy designation to NGN-401, Neurogene's gene therapy for Rett syndrome, based on interim data showing durable functional improvements. The company expects to complete pivotal trial dosing in Q2 2026.
Mar 04, 2026
New research demonstrates blood-based biomarkers can predict testicular cancer recurrence and treatment outcomes, while separate study reveals long-term health risks vary by chemotherapy regimen in survivors.
Mar 02, 2026
A CNIC study demonstrates that remote ischemic conditioning can protect the heart during anthracycline chemotherapy without compromising antitumor efficacy, while researchers explore additional methods to manage chemotherapy side effects.
Feb 17, 2026
New clinical trial data show trastuzumab deruxtecan plus pertuzumab extends progression-free survival as first-line therapy, while MRI-guided treatment shortening reduces toxicity in neoadjuvant settings for HER2-positive breast cancer.
