The diagnostics company BillionToOne has launched its first product for commercial and clinical use — a blood test designed to help diagnose hereditary and rare diseases in fetuses. Called UNITY, the ... Read more
Adding reldesemtiv to survival of motor neuron (SMN) restoration therapies — namely Spinraza (nusinersen) or the investigational SMN-C1 (an analogue to risdiplam) — can improve muscle function in a ... Read more
Optimal care of children with spinal muscular atrophy (SMA) requires improving competence and an adequate exchange of knowledge among healthcare professionals, as well maintaining a close dialogue with the ... Read more
The European Medicines Agency has granted a priority medicines, or PRIME, designation to oral therapy candidate risdiplam to treat spinal muscular atrophy (SMA). PRIME designation is intended to encourage the development of ... Read more
A non-invasive electrophysiology technique called motor unit number index, or MUNIX, can effectively assess the loss of motor nerve cells and muscle impairment in adults with spinal muscular atrophy ... Read more
Young children with spinal muscular atrophy show improvements in motor function after six-months of treatment with Spinraza, a German study shows.
The response to Spinraza strongly correlated with the age ... Read more
According to scientists, new research on ALS may shed light on the mechanisms underlying spinal muscular atrophy.
Two RNA binding proteins, TDP-43 and hnRNP A1, are abnormal in certain cases ... Read more
Researchers have uncovered the gene network regulating the transition of progenitor cells into motor neurons during the development of embryos, using chicken and mice as models. The study also ... Read more
Scholar Rock’s SRK-015 prevented additional atrophy in mice with muscle wasting and increased healthy animals’ muscle mass and function, a study reports. The biotech company’s therapy targets the precursor to the ... Read more
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