Spinal Muscular Atrophy and the possibilities with an investigational drug in a clinical trial
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 it harder 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 like onasemnogene abeparvovec (Zolgensma™), nusinersen (Spinraza™ ), and risdiplam (Evrysdi™), muscle weakness and fatigue remain ongoing challenges that can limit independence and make daily life more difficult.
If this sounds familiar, you’re not alone – and there is an investigational drug that could help.
NMD670 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 that is being evaluated in clinical trials in the US, Europe and Canada for people living with with spinal muscular atrophy (SMA). It is a skeletal muscle-targeted therapy that has 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 NMD670 and results demonstrated meaningful improvements in muscle strength for patients across multiple types of skeletal muscles (face, mouth/swallowing, arms, legs) throughout the body.1 These improvements remained improved at different timepoints through the day after taking the drug, but if patient was not taking drug (received placebo) the muscles were weaker at the later timepoints. Now, researchers are studying NMD670 in a clinical trial to understand the safety and efficacy of the drug in adults living with SMA. NMD670 is not expected to cure SMA, but it may provide meaningful improvements in muscle function making daily life easier and resulting in greater independence.
Join the Clinical Trial to Study the Potential Benefits and Safety of a New Investigational Skeletal Muscle-Targeted Drug
The SYNAPSE-SMA Study is evaluating an investigational drug called NMD670 in adults living with Type 3 spinal muscular atrophy (SMA) in clinical sites located in the US, Europe and Canada.
You may be able to take part in the SYNAPSE-SMA Study if you:
Are 18-75 years of age
Have been diagnosed with Type 3 SMA
Can walk at least 50 meters (164 feet) without a walking aid
Can attend 6 different in-clinic visits over a period of up to 13 weeks (depending on scheduling)
Taking part in this study is completely voluntary.
There is no cost to participate in this study, and your travel costs will be reimbursed (flight, mileage, hotel, parking, meals). Compensation may also be available for time taken for study visits or lost wages.
For more information and to request involvement in this important Adult SMA Clinical Trial, including cities/states of clinical trial sites and contact information for clinical coordinators, please visit clinicaltrials.gov or email NMD Pharma at: clinicaltrials@nmdpharma.com
NMD670 has not been approved by Regulatory Health Authorities and the safety and effectiveness of NMD670 in SMA, MG or CMT has not been established