Treatment with AJ201 was well tolerated and showed signs of improving physical activity in a clinical trial involving people with spinal and bulbar muscular atrophy (SBMA), according to new data announced by developer Annji Pharmaceutical. “I am greatly encouraged by the positive clinical outcomes observed after a relatively…

Training with a wearable robotic device called HAL, for hybrid assistive limb, improved walking ability for people with spinal muscular atrophy (SMA) who were also receiving treatment with Spinraza (nusinersen), according to a study in Japan. “[These] results may provide sufficient data to inform clinical decision-making about the…

Throughout 2024, SMA News Today brought our readers daily coverage of the latest clinical research and scientific breakthroughs related to spinal muscular atrophy (SMA). Here are the top 10 most-read stories we published last year, each with a brief description. We’re excited to continue serving the SMA community in…

NIDO-361, Nido Biosciences’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA) — a disease type marked by progressive weakness in the muscles of the throat and mouth, and those closer to the trunk — has been granted orphan drug designation in the European Union. This designation,…

Training with a wearable robotic hybrid assistive limb (HAL) device over two years was found to markedly improve the walking abilities of three patients with spinal and bulbar muscular atrophy (SBMA) who were also receiving treatment with leuprorelin, according to a new study by researchers in Japan. No notable…

A team of U.S. researchers has identified cellular mechanisms that might lead to the loss of nerve cell projections in spinal and bulbar muscular atrophy (SBMA), a rare adult-onset form of the genetic disease. That’s according to a new preclinical study showing that abnormal protein clumps in the cell’s…

Despite treatment with leuprorelin over three years, all spinal and bulbar muscular atrophy (SBMA) patients in a study in Korea continued to experience declines in motor function, a new study found. However, those individuals with less disability at the start of treatment experienced slower disease progression, “indicating a more…

A Phase 1b/2a clinical trial testing the experimental therapy AJ201 in people with spinal and bulbar muscular atrophy (SBMA) has finished enrolling patients and topline data are expected in the first half of 2024. The milestone was announced by Avenue Therapeutics, which last year acquired rights to…

Avenue Therapeutics has won the exclusive rights to develop and commercialize AJ201, AnnJi Pharmaceutical’s experimental treatment for spinal and bulbar muscular atrophy (SBMA), in certain regions of the globe under the terms of a new licensing agreement. Specifically, the agreement lists the U.S., Canada, the European Union,…