MASSACHUSETTS GENERAL HOSPITAL

A $75.4 million grant-funded clinical trial at UCSF will test three drugs concurrently for progressive supranuclear palsy (PSP), aiming to slow its progression. The trial, emphasizing diverse enrollment and offering financial support, uses an innovative platform model to efficiently test new therapies, offering hope for PSP patients.

Aficamten, a cardiac myosin inhibitor, improved exercise tolerance in patients with hypertrophic obstructive cardiomyopathy in the SEQUIOA-HCM trial, enhancing peak oxygen uptake and cardiac function, as published in JAMA Cardiology.

ASTRO announced seven studies to be highlighted at the 2024 Annual Meeting, with researchers discussing findings in news briefings on September 30 and October 1, both in-person and via webcast. Topics include trials on radiation therapy for breast cancer, prostate cancer, lung cancer, and oropharyngeal cancer, among others.

A $75.4 million NIH grant enables a UCSF-led clinical trial testing three drugs for progressive supranuclear palsy (PSP), aiming to slow its progression. The trial, involving up to 50 sites, uses a platform model to quickly identify effective therapies, prioritizing enrollment of diverse populations. CurePSP collaborates in recruitment, offering hope for patients and families.

New research shows low doses of carbon monoxide, similar to smoker exposure, protect against Parkinson's by preventing neurodegeneration and alpha-synuclein accumulation, suggesting further investigation into its disease-slowing potential.

ProJenX, Inc. forms Clinical Advisory Board (ClAB) with experts in ALS and neurodegenerative diseases to guide clinical development of prosetin, a novel brain-penetrant MAP4K inhibitor. Prosetin is being evaluated in Study PRO-101, a Phase 1 trial assessing safety and tolerability in healthy volunteers and ALS participants, with positive early results.

Vaderis announces positive results from its HHT clinical trial with VAD044, showing favorable safety, tolerability, and efficacy in key disease manifestations. Ongoing OLE data at 6 months confirm consistent safety and improvements in bleeding parameters.