NASHVILLE, Tennessee—While more new drugs for neuromuscular diseases are being approved than ever before, it’s still a challenge to make those therapies available to all patients who need them—and for doctors to match their patients to clinical trials as well as personalized care.

With that in mind, the Muscular Dystrophy Association (MDA) established its MOVR Data Hub in April 2019. An acronym for neuroMuscular ObserVational Research, MOVR gathers medical and genetic data through MDA’s network of 150 care centers from patients who agree to share their anonymized information.

“There’s a significant data shortage in the neuromuscular disease space,” said Rayne Rodgers, director of patient advocacy at Kinevant Sciences, and former vice president of MOVR. She was one of several officials who spoke at a panel on neuromuscular disease registry data at the MDA’s Clinical & Scientific Conference.

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Rodgers said MOVR now has 4362 patients enrolled across 7 diseases—up from around 2700 when it was launched. The current figure includes 1183 patients with Duchenne muscular dystrophy (DMD), 573 with spinal muscular atrophy (SMA), and 19 with Pompe disease.

“Clinical practices have changed even in the last year,” said Elisabeth Kilroy, PhD, the current director of MOVR, noting that when it comes to disease-modifying therapies in children, “the sooner we start these therapies, the better their outcomes.”

Likewise, the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) collects critical information about both DMD and Becker muscular dystrophy (BMD) to improve care for people living with these conditions.

Nicholas Johnson, MD, vice chair of research in neurology at Virginia Commonwealth University, Richmond, said MD STARnet was part of the 2001 MD-CARE Act authorized by Congress to create a national epidemiological surveillance program for muscular dystrophy.

Funded by the US Centers for Disease Control & Prevention, the network first surveyed DMD and BMD. Among other things, it found that the overall prevalence for DMD/BMD in 5 consecutive 5-year periods from 1991 to 2015 ranged from 1.9 to 2.4 per 10,000 boys aged 5-9 years.

Allison Moore, founder and CEO of the Hereditary Neuropathy Foundation, was diagnosed with Charcot-Marie-Tooth disease in 1997. In 2013, her nonprofit formed the Global Registry for Inherited Neuropathies (GRIN). That registry is now 100% virtual and is accessible via a mobile phone app, Moore said.

“We need to translate the patient voice into real-world data,” she said. “What’s really most important to patients is quality of life.”


Rodgers R, Kilroy E, Bétourné A, et al. Latest developments across the NMD registry data landscape. Oral presentation at: 2022 Muscular Dystrophy Association Clinical & Scientific Conference; March 14, 2022; Nashville, TN.