Background Major efforts have been made in the last decade to develop and improve
therapies for proximal spinal muscular atrophy (SMA). The introduction of …

Spinal muscular atrophy (SMA) linked to 5q is a recessive motor neuron disease
characterized by progressive and diffuse weakness and muscular atrophy. SMA is the most …

Spinal muscular atrophy (SMA) is a motor neuron disease caused by insufficient levels of
the survival motor neuron (SMN) protein. One of the most prominent pathological …

Objective Onasemnogene abeparvovec, a one-time intravenous gene replacement therapy,
and nusinersen, an antisense oligonucleotide that requires ongoing intrathecal …

Spinal muscular atrophy (SMA) is an uncommon disorder associated with genes
characterized by the gradual weakening and deterioration of muscles, often leading to …

Background and Objectives Spinal muscular atrophy (SMA) is a progressive neuromuscular
disorder associated with continuous motor function loss and complications, such as scoliosis …

Pediatric neuromuscular disorders are a diverse group of conditions that affect how muscle
and nerve function. They involve the motor neurons, nerves, neuromuscular junction and …

Methods Electronic databases were searched from inception to June 27, 2022 (Embase,
MEDLINE, and Evidence-Based Medicine Reviews). Observational studies, case-control …

Spinal muscular atrophy (SMA) is a monogenic disease that clinically manifests as severe
muscle weakness owing to neurotransmission defects and motoneuron degeneration …

Pediatric palliative care (PPC) is different from palliative care (PC) for adults. However,
conceptualizing PPC remains cumbersome due to the high heterogeneity of often rare …