At the all-virtual 2026 LGMD Scientific Summit, the development landscape for limb girdle muscular dystrophy ranged from early AAV gene-transfer studies reporting molecular and functional signals to a fully enrolled Phase III program moving into long-term follow-up. Together, these presentations showed that LGMD treatment is moving beyond a single measure of success.

Here is the medical news to watch for August 31 - September 6, 2026

Intravitreal gene therapy was a hot topic at ASRS this year. In age-related macular degeneration (wet AMD), the data looked promising for: 4D Molecular Therapeutics’ 4D-150 – the PRISM trial; Adverum Biotechnologies’ ixoberogene soroparvovec (ixo-vec) – the LUNA and OPTIC trials; REGENXBIO and AbbVie’s surabgene lomparvovec (sura-vec), and Elisigen and Reyon Pharmaceutical’s NG-101 – the SENSE trial. And in advanced geographic atrophy (GA), there were promising data for Astellas Pharma’s ASP-7317.

Less frequent dosing – both for PrEP and for HIV treatment – is on the horizon. Data presented at the International AIDS Conference suggest there may soon be: (1) A once every 6-month injectable for PrEP: Gilead Sciences’ Yeztugo (lenacapavir), and (2) A once-weekly oral for HIV treatment: Merck MSD’s islatravir + Gilead Sciences’ lenacapavir. However, the question is how long-interval dosing will be adopted, and if the experience with longer-acting treatments for HIV is a predictor, uptake may be slow.

Several studies were presented at AAIC that posed interesting questions: Is there a link between Covid-19 and Alzheimer’s disease? Is there a link between gabapentinoids and Alzheimer’s? Do GLP-1 RAs lower mortality in dementia patients? Can a blood test help diagnose Alzheimer’s? Are SQ injections better than IV infusions for treating dementia?

Posters presented at ASCP suggest psychiatry may be entering a new era of precision neuropharmacology – from new therapies for mood and anxiety disorders to psychedelics and psychedelic-like compounds.

The studies that caught our attention at the American Thoracic Society annual meeting ranged from a new use for a GLP-1 receptor agonist (obstructive sleep apnea, OSA), a potential new drug for OSA, a potential new drug to treat chronic rhinosinusitis with nasal polyps (CRSwNP), two drugs for chronic obstructive pulmonary disease (with results positive for one and mixed for the other), and a study that found “sleep divorce” may not always be a bad thing.

When the FDA speaks, it is a good idea to listen, and the annual FDLI conference offered a peek into the thinking of senior FDA officials – and the lawyers that have to deal with the Agency on a regular basis. Then FDA Commissioner Marty Makary and other senior FDA officials offered a look into the changes being initiated at the Agency.