SMA 特征性的呼吸系统病理生理改变主要为肋间肌无力、胸廓变形以及肺容量和通气能力降低，这些病理改变导致 SMA 患儿出现夜间和日间低通气、咳嗽减弱、痰液堵塞、反流与误吸、肺不张、反复呼吸道感染等呼吸系统问题[3]。

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...

After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation Patients continued to maintain and ...

The first program I want to go over is the use of taldefgrobep alfa, which is being developed in the phase 3 RESILIENT study for the treatment of patients with spinal muscular atrophy. Before going ...

The classification of spinal muscular atrophy (SMA) subtypes, the influence of SMN2 gene copies on disease severity, treatment effects on disease progression, age-related differences, long-term ...

First and only muscle-targeted SMA therapy to demonstrate statistically significant, clinically meaningful improvements on the gold-standard HFMSE scale (p=0.019) versus placebo, with consistent ...

Access to timely treatment for spinal muscular atrophy (SMA) remains a challenge, with barriers including delays in diagnosis, frequent treatment needs, and insurance coverage gaps, which impact ...