Muscular_atrophy,_spinal

Drug Candidate BIO101 for Spinal Muscular Atrophy as Monotherapy or Combined With the Antisense Oligonucleotide ASO-10-27.

<p><b>BACKGROUND</b></p><p>Spinal muscular atrophy (SMA) is a neuromuscular disease caused by loss of survival of motor neuron (SMN) protein inducing progressive muscle weakness and atrophy due to motor neurons degeneration. Despite benefits of SMN restoration therapies in patients, motor defects are...

๐Ÿ—“๏ธ 2025-10-23
๐Ÿ“ฐ Publication: Journal Of Cachexia Sarcopenia And Muscle
Read MoreDrug Candidate BIO101 for Spinal Muscular Atrophy as Monotherapy or Combined With the Antisense Oligonucleotide ASO-10-27.

Skeletal Muscle Mitochondrial and Autophagic Dysregulation Are Modifiable in Spinal Muscular Atrophy.

Spinal muscular atrophy (SMA) is a health- and life-limiting neuromuscular disorder. Although varying degrees of mitochondrial abnormalities have been documented in SMA skeletal muscle, the influence of disease progression on pathways that govern organelle turnover and dynamics are poorly understood....

๐Ÿ—“๏ธ 2025-02-01
๐Ÿ“ฐ Publication: Journal Of Cachexia Sarcopenia And Muscle
Read MoreSkeletal Muscle Mitochondrial and Autophagic Dysregulation Are Modifiable in Spinal Muscular Atrophy.

Feature Engineering for the Prediction of Scoliosis in 5q-Spinal Muscular Atrophy.

5q-Spinal muscular atrophy (SMA) is now one of the 5% treatable rare diseases worldwide. As disease-modifying therapies alter disease progression and patient phenotypes, paediatricians and consulting disciplines face new unknowns in their treatment decisions. Conclusions made from historical patient data...

๐Ÿ—“๏ธ 2024-12-05
๐Ÿ“ฐ Publication: Journal Of Cachexia Sarcopenia And Muscle
Read MoreFeature Engineering for the Prediction of Scoliosis in 5q-Spinal Muscular Atrophy.

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