A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been demonstrated by an analysis of data ...
"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...
Exploratory Phase 2a study showed consistent and clinically meaningful functional improvements across muscle strength, motor performance, and ...
- Taldefgrobep alfa, a myostatin-targeting biologic investigational agent, in Phase 3 development to increase muscle mass for Spinal Muscular Atrophy patients now granted Fast Track in addition to ...
Children with neuromuscular diseases and cerebral palsy often report disrupted sleep, which is usually attributed to breathing disorders. But new research points to a new potential contributor: ...
Spinal and bulbar muscular atrophy (SBMA) is a rare X-linked neuromuscular disorder that results from an abnormal expansion of gene sequences in the androgen receptor gene. SBMA has an adult onset and ...
Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. We are incredibly fortunate that the SMA ...
HOUSTON – A newly identified gene, atrogin-1, is involved in muscle loss associated with cancer, diabetes, fasting and kidney disease as well as in the atrophy occurring with disuse, inactivity, and ...
A phase 2a trial of NMD Pharma’s rare disease prospect ignaseclant has missed its primary endpoint. | A phase 2a trial of NMD ...
The struggle of a patient suffering from a rare, severe, and progressive neuromuscular genetic disorder has touched the heart of Health Minister Datuk ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results