The lab of Yongchao C. Ma, PhD, at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago uncovered a novel mechanism that leads to motor neuron ...

Spinal Muscular Atrophy (SMA) is a rare genetic disorder characterized by the progressive weakening of muscles due to the degeneration of motor neurons in the spinal cord. This condition is caused ...

Source Reference: Crawford TO, et al "Safety and efficacy of apitegromab in patients with spinal muscular atrophy types 2 and 3" Neurology 2024; DOI: 10.1212/WNL.000000000020915.

Spinal Muscular Atrophy. This is an inherited condition that affects lower motor neurons. A defect in a gene called SMN1 causes spinal muscular atrophy.This gene makes a protein that protects your ...

For spinal muscular atrophy (SMA), a progressive motor neuron disease, developing a gene therapy that edits the DNA may be the key to correcting an evolutionary genetic mistake. In patients with SMA, ...

Spinal muscular atrophy is a disease that is usually seen in babies and children. It makes their muscles weak and hard to move. ... Changes or defects in the SMN (survival motor neuron) ...

Treatment with nusinersen was linked to increased motor function and reduction of the neurodegenerative biomarker neurofilament light chain in children with spinal muscular atrophy, according to ...

Adult-onset spinal muscular atrophy occurs over the age of 18, and commonly in a person's 30s or 40s. ... SMN2 survival of motor neuron 2, centromeric [ Homo sapiens (human) ].

For the first time ever, doctors have treated a rare genetic disease known as spinal muscular atrophy (SMA) in the womb – and ...

Spinal muscular atrophy (SMA) is a genetic condition that leads to a progressive loss of nerve cells called “lower motor neurons” in the spinal cord.

The U.S. Food and Drug Administration (FDA) granted fast track designation to Biohaven (NYSE:BHVN) medicine taldefgrobep alfa to treat spinal muscular atrophy (SMA). SMA is an inherited motor ...

Covina, Feb. 15, 2024 (GLOBE NEWSWIRE) -- “According to the recent research study, the Spinal Muscular Atrophy Market size was valued at about USD 1.4 Billion in 2024 and expected to grow at ...