Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial to test its gene therapy candidate SRP-9003 in children with limb-girdle muscular dystrophy type 2E; wastewater testing points to a new COVID-19 infection wave fueled by the JN.1 variant; most patients on anti-obesity medications kept at least some weight off up to a year after they stopped taking them.
Phase 3 Trial of Gene Therapy in Children With LGMD2E Starts Screening
Sarepta Therapeutics has started screening participants for EMERGENE, a phase 3 clinical trial that will test its gene therapy candidate SRP-9003 in children with limb-girdle muscular dystrophy type 2E (LGMD2E), according to Muscular Dystrophy News. LGMD2E is caused by SGCB gene mutations, which limit the production of the muscle protein beta-sarcoglycan, resulting in an impaired sarcoglycan protein complex that leads to muscle weakness and wasting. SRP-9003 was designed to deliver a healthy version of the SGCB gene and increase beta-sarcoglycan levels in diaphragm, skeletal, and cardiac muscle cells. The trial’s main goal is to measure SGCB gene activity 60 days after dosing in 15 patients 4 years and older; it will also assess safety and functional parameters. The phase 3 trial follows promising results from a small phase 1/2 trial of 6 children with LGMD2E aged 4 to 13 yerars; SRP-9003 was well tolerated, resulting in a significant increase in beta-sarcoglycan levels in muscles after 2 years.
Wastewater Tests Show COVID Infections Surging Amid Less Severe JN.1 Variant Wave
Wastewater testing has indicated a new COVID-19 infection wave fueled by the JN.1 variant, with as many as one-third of Americans expected to contract the virus by late February, according to Stateline. Wastewater testing found that the current COVID-19 wave peaked in late December with 1.9 million daily infections, the highest since the omicron wave in 2021. Although the public has wearied of COVID-19 mitigation efforts, some experts want to maintain and expand wastewater surveillance to stay on top of future waves at local and state levels; they encourage people to avoid getting infected or reinfected with COVID-19 since every new infection increases one’s chances of long COVID. Conversely, CBS News reports that the CDC announced there is no current evidence that the JN.1 variant caused more severe disease. CDC official Eduardo Azziz-Baumgartner, MD, noted that the CDC hopes to release more details about JN.1’s severity sometime in “the next couple weeks” as more data accumulates, but it is unclear exactly when it will do so.
Many Patients Kept Weight Off After Stopping Anti-Obesity Med Use, Study Says
New data from Epic Research show that most patients on anti-obesity medications kept at least some weight off up to a year after they stopped taking them, according to Axios. The researchers looked at weight loss up to 12 months out from patients stopping the use of either liraglutide or semaglutide; they studied those prescribed the drugs for diabetes and those prescribed the drugs for weight loss. For example, of the 20,274 patients examined who lost at least 5 pounds while using semaglutide, nearly 20% regained all the weight they had lost or more and another 26% regained more than a quarter of their original weight but were short of total regain. Conversely, about 20% of patients essentially maintained their weight loss, as they did not regain or lose more than a quarter of their weight. Epic Research explained that their findings offer a more nuanced picture of patient outcomes.
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