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Clinical Trials

All articles tagged with #clinical trials

health1 day ago

Hope on Hold: DIPG Families Fight for Trials Amid U.S. Funding Cuts

A Guardian feature follows Izzy, a four-year-old with diffuse intrinsic pontine glioma (DIPG), and the families and doctors racing to find experimental treatments as the Trump era’s funding cuts cripple NIH support, shutter the Pediatric Brain Tumor Consortium, and suspend or delay dozens of clinical trials. The result is that progress in DIPG research stalls just as families face a last-hope window, turning access to new therapies into a bureaucratic and political obstacle. In the end, Izzy receives a reprieve when Stanford offers a CAR-T cell trial, a glimmer of hope amid a landscape of lost trials and dwindling resources.

via The Guardian|
#clinical-trials#dipg#health
health2 days ago

FDA Embraces Bayesian Toolkit to Accelerate Drug Trials

The FDA issued a draft guidance to promote the use of Bayesian methodologies in clinical trials for drugs and biologics, aiming to use prior information and data more efficiently to shorten timelines and reduce costs. The guidance outlines when Bayesian methods can support primary inference and trial design, including early futility/success decisions, dose selection, incorporating external data, enabling subgroup analyses, and aiding rare or pediatric studies; public comment is invited as part of PDUFA VII commitments.

via fda.gov|
#bayesian-methods#clinical-trials#drug-development
health3 days ago

Gene therapies promise cures, but getting them to patients remains a bottleneck

Gene therapies are moving from trials toward patient care, with breakthroughs like personalized CRISPR treatments and new gene-editing tools powering hope for many diseases; however, turning lab successes into approved, accessible medicines is hampered by regulatory, manufacturing, safety, and cost challenges, leaving access uneven despite progress.

via The Washington Post|
#biotechnology#clinical-trials#gene-therapy
healthcare11 days ago

Top Clinical Trials and Results to Watch in Early 2026

The article previews ten significant clinical trials expected in the first half of 2026, focusing on Eli Lilly's obesity drug retatrutide, Merck's flu antiviral CD388, Regenxbio's gene therapy for Duchenne muscular dystrophy, Novartis' Lp(a) cardiovascular treatment, and other innovative therapies for rare diseases and cardiovascular conditions. Success in these trials could lead to major advancements and market approvals in their respective fields.

via BioPharma Dive|
#antiviral#cardiovascular#clinical-trials
health13 days ago

Progress Nears in Developing Drug to Reduce Artery-Clogging Particle

Researchers are nearing the completion of clinical trials for five experimental drugs aimed at lowering lipoprotein(a), a genetic particle linked to artery clogging and heart disease, with results expected in 2026. These drugs could significantly impact the treatment of a condition affecting about 20% of the global population, though cost and public awareness remain challenges.

via The Washington Post|
#cardiovascular-disease#clinical-trials#experimental-drugs
health2 months ago

Swansea Boy, 4, Diagnosed with Rare Childhood Dementia

A 4-year-old boy from Swansea diagnosed with Sanfilippo Syndrome, a form of childhood dementia, has inspired a community fundraising effort to help him access clinical trials abroad, as there is currently no cure for his condition. His mother, Tammy McDaid, is focused on making memories and slowing the disease's progression, highlighting the urgent need for awareness and treatment options.

via BBC|
#childhood-dementia#clinical-trials#fundraising