A recent study published in the Journal of Neuromuscular Diseases has revealed that neuroMuscular ObserVational Research Data Hub (MOVR) can serve as a potential platform for informing disease understanding, clinical trial design guidance, and accelerating the drug development for neuromuscular diseases (NMDs).

According to the researchers, the MOVR platform collects longitudinal data from individuals diagnosed with various diseases, including amyotrophic lateral sclerosis (ALS), Becker muscular dystrophy (BMD), Pompe disease (PD), Facioscapulohumeral muscular dystrophy (FSHD), Duchenne muscular dystrophy (DMD), Limb girdle muscular dystrophies (LGMD), or spinal muscular atrophy (SMA), and individuals who are seen for care at the participating Muscular Dystrophy Association (MDA) care center.

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Using medical records, the data are entered into standardized electronic case report forms (eCRFs). These eCRFs then capture clinical visits, diagnostic journeys, discontinuation, and demographics data of participants from the study.

“The goal of MOVR is to efficiently capture clinical data from multidisciplinary visits happening throughout the MDA Care Center Network,” the authors wrote.

Between January 2019 and May 2022, data were gathered from 50 participating care centers and 1,957 participants. Moreover, data from 1,923 study cohort members who participated in the MDA’s pilot registry were migrated into MOVR.

Furthermore, the initial analysis of aggregated data showed that 91% of eCRFs were finalized, and 43% of participants reported 3 or more encounters, while 50% of all encounters were 5 months or less compared to the previous encounter.

“MOVR is a versatile and rigorous data platform designed to ensure that data from every clinic visit at participating MDA Care Centers are captured in a format that can be used for a variety of purposes,” the researchers noted.

Neuromuscular diseases (NMDs) encompass a range of rare and largely genetic conditions characterized by nerves and skeletal muscle dysfunction resulting in the progressive weakening of the nerves and muscles. Developing effective treatments for NMDs is a challenging task.

However, investments in basic research by public and private funding sources, including over $1 billion from the Muscular Dystrophy Association (MDA), have resulted in progress in drug development. In the past decade, 14 new therapies for NMDs have been approved, and more than 180 additional treatments are in various stages of development.

Statistics have revealed that in the EU, using registries from 2000 to 2019 was a crucial element in the approval process for almost 90% of conditionally approved orphan drugs and all those approved under “exceptional circumstances.” Consequently, it is now more important than ever to collect and analyze clinical data for NMDs before and after treatment to ensure regulatory compliance and continued access to effective medications for patients.

The pilot program of the United States Neuromuscular Disease Registry has demonstrated success and value in the NMD field, leading to the launch of MOVR in 2019. This updated platform streamlines data entry and validation and covers 7 indications across 50 sites.


Kilroy EA, Burris R, Javelosa E, et al. The Muscular Dystrophy Association’s neuromuscular observational research data hub (MOVR): design, methods, and initial observations. J Neuromuscul Dis. Published online March 7, 2023. doi: 10.3233/JND-221551