Current Trials

Our current research targets the following disorders.

• Muscular Dystrophy
• Spinal Muscular Atrophy
• Pediatric Epilepsy

How do I know if I am eligible to participate in a clinical trial?

Each study has a different purpose and specific scientific questions that it is trying to answer. In order to achieve this, very specific criteria, or requirements, are developed to evaluate an individual’s ability to participate. These criteria will determine if you are a good candidate for inclusion, or if there is any reason that you should be excluded from the study (such as due to other health conditions). After agreeing to be assessed for participation, you will undergo a very detailed screening process at no cost to you, to determine your eligibility to participate in a study.

Our novel investigator-initiated clinical research studies include.

• Development of improved rating scales and outcome measures for rare diseases, which can improve the likelihood that effective interventions will successfully transition to FDA-approved treatments
• Testing of biomarkers that are associated with clinically meaningful changes in studies of new therapeutics 
• Development of newborn screening strategies for rare genetic diseases 
• Testing and utilizing novel, wearable technology designed to measure changes in physiologic function relevant to rare disease research and therapeutic management