Results 211 to 220 of about 1,032,826 (402)

The Role of Repeat Skeletal Muscle Biopsy: Indications, Yield and Outcomes

open access: yesMuscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Muscle biopsy performed to investigate weakness and/or pain may be nondiagnostic and prompt repeat biopsy. We determined the indications and yield of rebiopsy. Methods Patients who underwent > 1 muscle biopsy (South Australia, 2000–2023) were identified.
Thomas Khoo   +4 more
wiley   +1 more source

A Phase 1, Double‐Blind, Placebo‐Controlled Trial of Sevasemten (EDG‐5506), a Selective Modulator of Fast Skeletal Muscle Contraction, in Healthy Volunteers and Adults With Becker Muscular Dystrophy

open access: yesMuscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Sevasemten (EDG‐5506) is an orally administered, investigational small molecule that selectively modulates fast muscle fiber contraction by inhibiting fast myosin ATPase. This study assessed the safety, tolerability, and pharmacokinetics (PK)/pharmacodynamics (PD) of sevasemten in healthy adult volunteers (HVs) and adults ...
Joanne Donovan   +11 more
wiley   +1 more source

Serum Cardiac Troponin T Levels as a Therapy Response Marker in Tofersen‐Treated ALS

open access: yesMuscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Cardiac troponin T (cTnT) levels are elevated in the majority of persons with amyotrophic lateral sclerosis (ALS) and increase over time. Neurofilament light chain (NfL) is an established therapy response biomarker in ALS as superoxide dismutase1 (SOD1)‐ALS patients treated with the antisense oligonucleotide tofersen show a ...
Sarah Bernsen   +7 more
wiley   +1 more source

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