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…
The life expectancy for people with SMA depends on the type of their disease and symptom severity. Starting treatment early is thought to improve life expectancy, and disease-modifying therapies can work to slow or halt progression of the various symptoms of SMA.
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,…
A gene-silencing therapy designed to lower the levels of two proteins was found to improve muscle strength and coordination in a mouse model of spinal and bulbar muscular atrophy (SBMA), a new study shows. The therapy, which targets the protein lysine-specific demethylase 1 (LSD1) and the protein arginine methyltransferase…
Men with spinal and bulbar muscular atrophy (SBMA) had slower nerve impulses leading to their forearm muscles when their arms were exposed to cold temperatures, a small study reported. These slower impulses correlated with reduced hand grip strength, and were significantly longer in adults with muscle weakness at cold…
Moderate-intensity resistance training led to gains in muscle strength, balance and functional improvements in a man with mild impairments due to spinal and bulbar muscular atrophy (SBMA), according to a recent case report. “This case report highlights one individual with SBMA who benefitted from moderate-intensity exercise, including weight lifting,…
A new mutation in the UBEA1 gene was identified as the cause of an atypical form of spinal and bulbar muscular atrophy (SBMA), or Kennedy’s disease, in four men of an Iranian family, a study shows. While UBEA1 mutations are typically associated with X-linked infantile spinal muscular atrophy (SMA), which is…
The RNA-binding protein Mid1 increases levels of the mutated androgen receptor (AR) that drives nerve cell degeneration in spinal and bulbar muscular atrophy (SBMA), a new study shows. Since Mid1 is expressed mainly in lower motor neurons, the nerve cells mainly affected in SBMA, the study’s researchers proposed that…
Two years of training with a wearable cyborg hybrid assistive limb (HAL), combined with leuprorelin therapy, markedly improved the walking abilities of a man with spinal and bulbar muscular atrophy (SBMA), a case study reports. “This report describes the first successful case in the history of SBMA treatment in…
Blood biomarker levels of muscle damage — rather than nerve cell damage — are significantly altered in people with a rare form of adult-onset spinal muscular atrophy (SMA) called the Jokela type (SMAJ), according to a study in Finland. Also, the levels of creatinine, a biomarker of muscle mass,…