Featured Articles
Care Across the Lifespan
The patient journey starts with a diagnosis and often involves pediatric-to-adult transition, clinical follow-ups and multidisciplinary disease management. In recent years, the care model for neuromuscular disease has evolved into a patient-centric treatment approach, in which individuals are driving their own care decisions.
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From Drug Discovery to Delivery
MDA was honored to have Janet Woodcock, M.D., director of the Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration (FDA), present the keynote address at MDA’s 2019 Clinical & Scientific Conference. Her department, the CDER, oversees most human trials with investigational drugs — those not yet approved or not approved for a particular indication —– and conducts post-market safety surveillance for all drugs on the market to see if new safety findings occur once the drug is being widely used.
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The State of Gene-Targeted Therapies
The first full day of MDA’s Clinical & Scientific Conference included a highly anticipated session that took a deep dive into gene-targeted therapies. Experts discussed how gene-replacement, gene-silencing and gene-editing therapies are being studied and tested for treating patients with a variety of neuromuscular diseases (NMDs).
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Ongoing Clinical Trials
During the second day of the 2019 MDA Clinical & Scientific Conference, 14 scientists and clinicians shared exciting updates from the pipeline of clinical trials. Below are highlights from the session.At the time of the conference, Zolgensma (onasemnogene abeparvovec-xioi, or AVXS-101) was under review by the U.S. Food and Drug Administration (FDA). In May, the FDA approved the drug as a therapy for the treatment of children younger than age 2 with spinal muscular atrophy (SMA).
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The Case for Newborn Screening
Newborn screening allows babies born with life-threatening diseases to be treated before they show any signs of disease, which can lead to improved outcomes and maybe even a life free of symptoms.As therapies for neuromuscular diseases progress through the clinical pipeline and get approved by the U.S. Food and Drug Administration (FDA), the importance of newborn screening is heightened. Currently, it is recommended that states screen for Pompe disease and spinal muscular atrophy (SMA), and a screening for Duchenne muscular dystrophy (DMD) could be on the horizon. But despite these advancements, some states face implementation challenges due to scientific, economic and operational considerations.
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