Please reach out to the Research Coordinator listed as the contact for studies of interest to determine if they are currently enrolling.
All Neuromuscular Disorders
MD STARnet
Population: Adult and Pediatric
Disease: All Neuromuscular
Enrolling: Not at this time
Muscular Dystrophy Surveillance, Tracking and Research Network.
For more information, contact Research Coordinator Sarah Moldt at: sarah.moldt@hsc.utah.edu
PSYQ-MD
Population: Adult and Pediatric
Disease: All Neuromuscular
Enrolling: Yes
More information to come.
For more information, contact Research Coordinator Sarah Moldt at: sarah.moldt@hsc.utah.edu
Amyotrophic Lateral Sclerosis (ALS)
GTAC
Population: Adult
Disease: Amyotrophic Lateral Sclerosis (ALS)
Enrolling: CLOSED
Genomic Translation for Amyotrophic Lateral Sclerosis (ALS) Care.
For more information, contact Research Coordinator Crystal Neate at: crystal.neate@hsc.utah.edu
MTPA REFINE-ALS
Population: Adult
Disease: Amyotrophic Lateral Sclerosis (ALS)
Enrolling: Yes
An observational study exploring biomarkers associated with Edaravone use in ALS patients.
For more information, contact Research Coordinator Mike Papadakis at:
Collagen VI
COL VI
Population: Adult
Disease: Collagen VI
Enrolling: Yes
Clinical and Molecular Characterization of Collagen VI Myopathies.
For more information, contact Research Coordinator Sarah Moldt at: sarah.moldt@hsc.utah.edu
Duchenne Muscular Dystrophy (DMD)
D3 Creatinine DMD Biomarkers Project
Population: Pediatric
Disease: Duchenne Muscular Dystrophy (DMD)
Enrolling: Not at this time
More information to come.
For more information, contact Research Coordinator Shelby Murdock at: shelby.murdock@hsc.utah.edu
Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHD ReSolve
Population: Adult
Disease: Facioscapulohumeral Muscular Dystrophy (FSHD)
Enrolling: Not at this time
Clinical Trial Readiness to Solve Barriers to Drug Development in Facioscapulohumeral Muscular Dystrophy.
For more information, contact Research Coordinator Sarah Moldt at: sarah.moldt@hsc.utah.edu
FSH Wellstone
Population: Adult
Disease: Facioscapulohumeral Muscular Dystrophy (FSHD)
Enrolling: Yes
We are interested in following up with Utah families who have a history of FSHD to identify genetic variants that influence the severity of disease. You can take our study survey HERE!
For more information, contact Research Coordinator Sarah Moldt at: sarah.moldt@hsc.utah.edu
MOVE FSHD
Population: Adult
Disease: Facioscapulohumeral Muscular Dystrophy (FSHD)
Enrolling: Not at this time
More information to come.
For more information, contact Research Coordinator Sarah Moldt at: sarah.moldt@hsc.utah.edu
Limb Girdle
Sarepta JOURNEY (SRP-LGMD-NHS)
Population: Adult
Disease: Limb Girdle
Enrolling: Not at this time
More information to come.
For more information, contact Research Coordinator Shelby Murdock at:
Leukodystrophy
Swan Bio
Population: Pediatric
Disease: Leukodystrophy
Enrolling: Not at this time
A prospective, retrospective, multicenter, observational study of disease progression in adult with inherited forms of Spastic Paraplegia. Progressive weakness and spasticity of the legs are characteristics of numerous disorders and conditions, including those that are inherited neurological disorders. Adrenomyeloneuropathy (AMN) is an example of an inherited form of spastic paraplegia. This study will monitor participant’s PT assessments and quality of life questionnaires for two years to assess the needs of those with AMN.
For more information, contact Research Coordinator Kelsee Parry at:
GLIA-CTN
Population: Pediatric
Disease: Leukodystrophy
Enrolling: Yes
The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trails Network – The primary purpose of this study is to establish new tests to diagnosis Leukodystrophies and understand how Leukodystrophy progress over time in people.
For more information, contact Research Coordinator Tate Keough at:
Myotonic Dystrophy
Cognitive Function & Neuroimaging in DM1
Population: Pediatric
Disease: Myotonic Dystrophy (DM)
Enrolling: Yes
This study aims to identify differences in brain connectivity in children with congenital myotonic dystrophy (CDM) and childhood-onset myotonic dystrophy (chDM1) compared to controls that can be measured at baseline and one year, as well as demonstrate that clinical outcomes of cognitive function correlate with brain connectivity and network changes.
For more information, contact Research Coordinator Becky Crockett at: bcrockett@genetics.utah.edu
Spinal Muscular Atrophy
Avexis 101 Registry
Population: Pediatric
Disease: Spinal Muscular Atrophy (SMA)
Enrolling: Yes
A Long Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy.
For more information, contact Research Coordinator Shelby Murdock at: shelby.murdock@hsc.utah.edu
Cure SMA Registry
Population: Pediatric
Disease: Spinal Muscular Atrophy (SMA)
Enrolling: Yes
A Multicenter Cure SMA Clinical Data Registry.
For more information, contact Research Coordinator Sarah Moldt at: sarah.moldt@hsc.utah.edu