Self-Assessment: Add-on Therapy for SMA | Navigating Through the Available Therapies for Duchenne: How Do You Choose?-Add-On Therapy to SMA: Dr. Cardon Presentation

Add-On Therapy to SMA: Dr. Cardon Presentation

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The presentation by Meeta Cardon, MD, discusses the use of myostatin inhibitors for treating patients with Spinal Muscular Atrophy (SMA), the most common motor neuron disease in children. While FDA-approved treatments exist, they do not completely alleviate all symptoms, highlighting the need for additional therapies. Myostatin, a negative regulator of skeletal muscle growth, represents a potential target for enhancing muscle development in SMA patients.<br /><br />Several strategies for myostatin inhibition were explored, including Apitegromab, a human monoclonal antibody binding to myostatin, and Taldefgrobep, an anti-myostatin adnectin protein. These inhibitors have shown promising results in animal models, increasing muscle weight and function. Notably, Apitegromab was evaluated in the TOPAZ study, demonstrating some potential to increase muscle mass and improve motor skills when combined with nusinersen, another SMA therapy.<br /><br />The clinical trials, such as the TOPAZ trial and the phase 3 RESILIENT trial, are investigating these inhibitors' efficacy and safety in various cohorts of patients with SMA. GYM329, another myostatin inhibitor, is being tested in the MANATEE trial, further exploring its potential in combination with risdiplam, a treatment for SMA.<br /><br />The presentation emphasizes the innovative forms of myostatin inhibitors, including sweeping antibodies and human monoclonal antibodies, which may have fewer off-target effects and could effectively treat muscle wasting in SMA. While advancements have been made, ongoing clinical trials are crucial to determining efficacy, safety, and optimal use of these treatments for SMA patients. Attendees are encouraged to provide feedback via the AANEM app to enhance future presentations and ensure they continue to meet participant needs.

Keywords

Myostatin inhibitors

Spinal Muscular Atrophy

SMA

Apitegromab

Taldefgrobep

TOPAZ study

Clinical trials

Muscle growth

Motor neuron disease

MND

GYM329

motor neuron disease

MND

spinal muscular atrophy

SMA