Spinal Muscular Atrophy
Living with Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a rare, inherited condition that affects the nerves that control muscle movement. Adults living with SMA experience muscles that feel weak and tire easily, especially in the legs and arms. This can make life frustrating and even impossible to do everyday things like walk, climb stairs, lift objects or raise up from a chair. Even with the exciting advances in SMN-targeted treatments, muscle weakness and fatigue remain ongoing challenges that can limit independence and make daily life more difficult.
If this sounds like your experience, you’re not alone - and an investigational drug is being studied to help improve muscle strength and endurance.
NMD Pharma’s Treatment Approach: Targeting the Skeletal Muscles Affected by SMA
Ignaseclant is an investigational drug in a new class of treatment, featuring a novel mechanism of action distinct from current SMN-targeted therapies or other muscle directed therapies. It is an oral medication taken with a simple glass of water.
Ignaseclant is being evaluated in clinical trials in the US, Europe and Canada for adults living with spinal muscular atrophy (SMA). It is a skeletal muscle-targeted therapy that is being investigated for the potential to increase muscle responsiveness to weak nerve signals and enable more consistent muscle function.
In a previously completed, early phase placebo-controlled clinical trial in a different neuromuscular disease also characterized by weak nerve signals reaching the muscles, patients received only a single dose of ignaseclant and results indicated meaningful improvements in muscle strength for patients across multiple types of skeletal muscles (face, mouth, swallowing, arms, legs) throughout the body.1 Now, researchers are studying ignaseclant in a clinical trial to understand the safety and efficacy of the drug in ambulatory adults living with SMA. Though ignaseclant is not expected to cure SMA, it may provide meaningful improvements in muscle function making daily life easier and resulting in greater independence.
1 https://www.nature.com/articles/d41573-024-00065-9
Ignaseclant has not been approved by Regulatory Health Authorities and the safety and effectiveness of ignaseclant in SMA, MG or CMT has not been established