Frontiers3d
Clinical Phenotyping and Biomarkers in Spinal and Bulbar Muscular Atrophy
Background: Spinal and bulbar muscular atrophy (SBMA) or Kennedy disease [OMIM: 313200] is a rare X-linked neuromuscular disease. Patients commonly present with muscle cramps, tremors, leg weakness, ...
Frontiers27d
Spinal muscular atrophy type 1 in the Caribbean: the first case report from the Dominican Republic
Spinal muscular atrophy (SMA) is a progressive genetic neuromuscular condition ... Genetic testing has been requested due to the patient’s current clinical history and family background, confirming a ...
WUWM27m
Spinal stimulation restored muscles wasted by rare genetic disorder
Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal ...
Healio3d
FDA receives biologics license application for apitegromab in spinal muscular atrophy
A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...
The Hindu29d
14-year-old author with Spinal Muscular Atrophy inspires others with her resilience and determination
who are living with Spinal Muscular Atrophy (SMA) with the support of her mother, sister and teacher. Ms. Keya was felicitated by the Visakha Rasagna Vedika, a cultural organisation, at the Public ...
The Hindu29d
Experts seek affordable access to medicines for treating Spinal Muscular Atrophy
Seeking urgent Union government intervention to overcome the lack of affordable access to medicines for treating Spinal Muscular Atrophy (SMA) and the need to allow local manufacturers to make ...
WebMD8d
Living Long-Term With SMA
So the outcome and the long-term outlook for our patients with SMA, particularly in the adult populations are that they are now stronger and living longer. And so as a result of that, they're able ...
contemporarypediatrics13d
FDA accepts sNDA for higher regimen nusinersen for spinal muscular atrophy
The FDA has accepted a supplemental New Drug Application for a higher dose regimen of nusinersen (Spinraza; Biogen Inc) for spinal muscular atrophy (SMA), according to a recent announcement from ...
Pharmaceutical Technology18d
Talditercept alfa by Biohaven for Spinal Muscular Atrophy (SMA): Likelihood of Approval
Talditercept alfa is under clinical development by Biohaven and currently in Phase III for Spinal Muscular Atrophy (SMA). According to GlobalData, Phase III drugs for Spinal Muscular Atrophy (SMA) ...
BMJ28d
Respiratory management of the infant with type 1 spinal muscular atrophy
NIPPV, nasal intermittent positive pressure SMA-1, type 1 spinal muscular atrophy Some preliminary observations are pertinent. The first is that the prognosis of children with neuromuscular disease ...
SMA27d
Exercise and physical therapy for SMA
Exercise can enhance the lives of people with spinal muscular atrophy (SMA) by improving or maintaining function and quality of life. This is accomplished through activities that help to maintain or ...