MDA Clinical & Scientific Conference 2024 Highlights
Challenges and Opportunities in the Treatment of Neuromuscular Disease in the Age of Therapies
The fast-paced development and approval of therapies for the treatment of neuromuscular diseases (NMDs) have reshaped the landscape of neuromuscular health care over the past decade. Experts in precision medicine, health care systems, drug development, and patient care discussed the challenges and opportunities associated with novel therapies, as well as the need for collaboration and personalized care, in a general session at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida.
March 5, 2024
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Families Affected by Progressive Neuromuscular Disorder Hold on to the Promise of Multiple Therapies
Evolving therapeutic options for spinal muscular atrophy (SMA) have provided hope for affected individuals and their families, slowing disease progression, helping patients achieve new milestones, and prolonging survival.
March 6, 2024
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Individuals with Spinal Muscular Atrophy Treated with Survival Motor Neuron-Targeted Therapies Struggle with Fatigue and Quality of Life
Disease-modifying therapies have opened new avenues for individuals with SMA, improving survival, disease course, and motor function. Despite significant advances in therapy, many people affected by this condition continue to experience reduced quality of life and limited mobility.
March 5, 2024
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Early Treatment with Risdiplam Provides Significant Benefit for Infants with Spinal Muscular Atrophy
A majority of infants with presymptomatic spinal muscular atrophy (SMA) treated with risdiplam achieved important motor milestones after 12 months of treatment without experiencing significant adverse events, according to data from the RAINBOWFISH trial presented at the 2024 MDA Clinical and Scientific Conference in Orlando, Florida.
March 5, 2024
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Overcoming Barriers to Transition from Pediatric to Adult Neuromuscular Health Care
At an age when young adults undergo many biological and psychosocial changes, individuals living with neuromuscular diseases (NMDs) also face the hurdle of transitioning from pediatric chronic care to the specialized services of adult health care providers. “This is a tricky area and age,” said Amanda Witt, MD, Professor of Neurology at the University of Mississippi Medical Center, in Jackson, Mississippi.
March 5, 2024
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Neuromuscular Care Takes a Village: Multidisciplinary Approaches Can Optimize Treatment of Adults with Neuromuscular Disorders
As individuals with neuromuscular disorders (NMDs) navigate an increasingly complex clinical reality that has been reshaped by genetic testing and novel therapies, many can benefit from a multidisciplinary approach to neuromuscular care. This approach, which has been successfully implemented at the Vanderbilt University Neuromuscular Disorders Clinic, in Nashville, Tennessee, can facilitate access to care for patients by meeting all their health care needs in one place.
March 6, 2024
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Early Combination Therapy May Benefit Children with Spinal Muscular Atrophy
The availability of multiple disease-modifying therapies (DMTs) for the treatment of spinal muscular atrophy (SMA) has altered the course of disease and prognosis for many individuals living with this condition.
Mar 6, 2024
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Physical Therapy Is Underutilized in Children and Adults with Spinal Muscular Atrophy
Individuals with spinal muscular atrophy (SMA) benefit from an approach to care that addresses not only their symptoms but also the various functional limitations associated with the loss of motor neurons.
March 6, 2024
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Understanding the Risks of Gene Transfer Therapies Can Improve Safety of Patients with Neuromuscular Disorders
While gene replacement therapies have expanded the armamentarium for the treatment of neuromuscular disorders (NMDs), these novel interventions may sometimes result in unwanted outcomes.
March 7, 2024
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Monitoring Patients After Gene Transfer Therapies Is an Important Link in the Chain of Neuromuscular Care
Gene transfer therapies have improved the odds of living a life with reduced or no disability for many patients with neuromuscular disorders (NMD).
March 7, 2024
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Evaluation of the Cognitive Impact of Spinal Muscular Atrophy Requires Validated Instruments
Spinal muscular atrophy (SMA) is a congenital motor neuron disease that causes progressive muscle weakness and disability. In studying SMA, researchers have focused mostly on the evaluation of motor function.
March 7, 2024
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Children with Spinal Muscular Atrophy May Benefit from Increased Access to Second Therapies
Individuals with spinal muscular atrophy (SMA) who have a suboptimal response to initial gene transfer therapy may benefit from subsequent treatment with a different agent, according to data presented at the 2024 MDA conference.
March 7, 2024
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