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Mar. 24, 2021 — Capturing the structure of large molecular complexes with variable shape is an extremely difficult task. Scientists now have been able to do it - thanks to a new approach regarding an important ...
Dec. 7, 2023 — Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes ...
July 13, 2022 — In 2016, Spinraza became the first FDA-approved treatment for spinal muscular atrophy. Researchers have found a way the therapeutic effects of Spinraza may be improved. They discovered that pairing ...
Mar. 14, 2022 — This study's findings will help congenital neurological disease (e.g. spinal muscular atrophy) specialists better understand the mechanisms and components involved in CNS development. Notably, ...
Sep. 1, 2021 — Researchers successfully developed and validated a new outcome measure to monitor disease severity and progression in patients with impaired skeletal muscle function caused by mitochondrial disease. ...
Apr. 11, 2022 — Patients who took part in the study saw a 1.6-kilogram increase in their muscle mass and a two per cent reduction of body fat. They were also able to walk an extra 47 meters in six minutes, when ...
June 20, 2023 — Scientists have written a review to discuss emerging research and evidence of the roles of alternative splicing defects in major neurodegenerative diseases. They also summarize the latest advances in ...
Nov. 3, 2022 — A new collaborative study shows how blood vessel genes play a critical role in motor neuron development by telling blood vessels to get out of the way. The discovery has implications for ...
July 14, 2022 — Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Scientists have discovered a new way ASOs may help cells produce a protein missing in cystic ...
Nov. 30, 2021 — Researchers have confirmed a link between altered DNA repair and increased DNA damage associated with spinocerebellar ataxia type 7 (SCA7), a debilitating, sometimes deadly neurodegenerative ...
