Sarepta’s Duchenne Gene Therapy Does Not Have ‘Unambiguous Evidence,’ FDA Staff Says
Clinical studies to date “do not provide unambiguous evidence” for SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for ambulatory Duchenne muscular dystrophy patients, FDA reviewers said in briefing documents released ahead of an advisory committee meeting. The agency also raised safety concerns about the possibility of administering an ineffective gene therapy. Duchenne muscular dystrophy is […]
Snipless Vasectomy Shows Early Promise as Long-Term Male Contraceptive
CHICAGO — A no-scalpel vasectomy passed a preliminary clinical test, achieving near-100% azoospermia within 2 weeks and maintaining the status to day 30 in the first men to undergo the procedure. Following injection of an occlusive nonhormonal hydrogel into the vas, sperm concentration decreased by as much as 99.98% by day 14 in the first […]
Clinical Note Writing App Powered by GPT-4 Set to Debut This Year
The first fully automatic clinical documentation application powered by OpenAI’s GPT-4 is scheduled to be widely released later this year, Nuance and Microsoft announced. This latest artificial intelligence (AI)-powered application, known as Dragon Ambient eXperience (DAX) Express, will be able to create an automatic clinical note based on recordings of patient visits, said Diana Nole, […]
Lone star tick bites may be to blame for unexplained digestive problems
The American Gastroenterological Association (AGA) has released new clinical guidance to help physicians and patients identify if unexplained digestive symptoms are due to alpha-gal syndrome, a food allergy that is caused by lone star tick bites. The AGA Clinical Practice Update was published today in Gastroenterology. Alpha-gal syndrome is an allergy that causes your body […]
Sotatercept, Fixed-Dose Combo Therapy on the Right Path for PAH
NEW ORLEANS — The limited clinical evidence thus far supports investigational compounds for addition to or simplification of existing frontline therapies for pulmonary arterial hypertension (PAH), according to two trials presented here at the American College of Cardiology (ACC) annual meeting. The activin signaling inhibitor biologic sotatercept showed promise as an add-on to usual treatment […]
Opinion | ‘It Verges on Malpractice’: What We Heard This Week
“It verges on malpractice.” — Adrian Hernandez, MD, of the Duke Clinical Research Institute in Durham, North Carolina, commenting on a MedPage Today investigative report that showed Texas physician Stella Immanuel, MD, was the nation’s top prescriber of hydroxychloroquine and ivermectin over the past 2 years. “We have a bad disease, we have a good […]
Bempedoic Acid Proves Clinical Benefits for the Statin-Intolerant
NEW ORLEANS — Bempedoic acid (Nexletol) provided compelling clinical benefit in terms of primary and secondary cardiovascular prevention for statin-intolerant patients who need cholesterol lowering, according to the CLEAR Outcomes trial. Cementing its place in the arsenal of nonstatin therapies, the ATP citrate lyase inhibitor met the primary endpoint in reducing major adverse cardiovascular events […]
CDC Offers Tentative Guidance for Severe Mpox Treatment
With no clinical trial data yet available for treating severe mpox, the CDC has published an interim report with guidance. There are many FDA-regulated drugs and biologics stockpiled for smallpox preparedness that can be used to treat severe mpox; however, there are many clinical considerations when using these to help manage strategic decision-making, the CDC […]
Scattered Brain Bleeds, Vasculitis in Alzheimer’s Trial Death
A fatal bleeding event in an Alzheimer’s clinical trial participant may have been linked with the investigational anti-amyloid agent lecanemab, a case report suggested. However, drug trialists are not willing to assign blame yet, especially as lecanemab’s fate hangs in a potential FDA approval for early Alzheimer’s disease later this week. Information about the case […]
Dementia Risk Score Touts ‘Nearly 100%’ Predictive Accuracy
A novel clinical risk score predicted the chance of future dementia with high accuracy, but raised questions from dementia experts. Using data from nearly 450,000 people in the U.K. Biobank cohort, Xi-jian Dai, PhD, of The Second Affiliated Hospital of Nanchang University in China, and colleagues developed a practical tool to predict individual risk of […]