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Muscle atrophy is the loss of skeletal muscle mass. It can be caused by immobility, ... Given the implications of muscle atrophy and limited treatment options ...
Progressive muscular atrophy (PMA), also called Duchenne–Aran disease and Duchenne–Aran muscular atrophy, is a disorder characterised by the degeneration of lower motor neurons, resulting in generalised, progressive loss of muscle function.
Nusinersen, [7] marketed as Spinraza, [4] is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. [8] [4] In December 2016, it became the first approved drug used in treating this disorder.
People with spinal muscle atrophy (SMA), an inherited neuromuscular disease, usually experience muscle weakness that impacts movement. New research suggests that electrical spinal cord stimulation ...
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle wasting. [ 3 ] [ 4 ] [ 5 ] It is usually diagnosed in infancy or early childhood and if left untreated it is the most common genetic cause of infant death. [ 6 ]
Spinal and bulbar muscular atrophy (SBMA), popularly known as Kennedy's disease, is a rare, adult-onset, X-linked recessive lower motor neuron disease caused by trinucleotide CAG repeat expansions in exon 1 of the androgen receptor (AR) gene, which results in both loss of AR function and toxic gain of function.
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), [6] [7] a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. [6]
The first three such treatments were approved in 2017, launching at prices of $370,000 or more. Luxturna, a gene therapy for a rare disorder that causes vision loss, costs $425,000 per eye.