Author Archives: Andrea Lobo

Scientists at The Jackson Laboratory — a U.S.-based biomedical research nonprofit — were granted $22.8 million by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), for a novel method to develop gene editing therapies for neurological disorders, including…

Treatment with Spinraza (nusinersen) for two years led to improvements in apnea and oxygen saturation during sleep in children with spinal muscular atrophy (SMA), a real-world study in Australia reported. The treatment also contributed to ceasing ventilation support in some patients with less severe disease, and stabilized…

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…

TREAT-NMD and Aetion have formed a partnership to deliver real-world evidence and promote the development of new treatments for people with rare neuromuscular diseases, including spinal muscular atrophy (SMA). The projects will combine the expertise of the TREAT-NMD global registry network, which collects de-identified data from…

A 23-day-old girl with spinal muscular atrophy (SMA) type 2, later treated with Spinraza (nusinersen), underwent successful surgical repair of heart defects, according to a new case report. The researchers noted that individuals with SMA may be perceived as risky surgical candidates, considering the poor prognosis for…

Disease-modifying therapies (DMTs) used for spinal muscular atrophy (SMA) may contribute to the development of therapies for age-related progressive loss of muscle mass and strength, or sarcopenia, new research suggests. The survival muscle neuron (SMN) protein, whose deficiency causes SMA, was found at progressively lower levels with aging…

Microvasculopathy — damage to small blood vessels — occurs in patients with spinal muscular atrophy (SMA) and it can be made better by systemic SMN-restoring treatment, a new study reports. Survival motor neuron protein (SMN) is the protein that is defective in SMA. The available SMA treatments increase…