What the trial was testing
The trial enrolled 46 patients with muscular dystrophy. The study was sponsored by ReveraGen BioPharma, Inc. and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Boys on vamorolone grew normally while maintaining muscle strength, unlike standard steroid treatment which slows growth.
JAMA network open · 2022 · NCT03038399
These findings — that boys maintained their height percentiles instead of falling behind in growth — were published in the JAMA network open and represent the headline result of the study.
Researchers tracked outcomes across 46 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with muscular dystrophy, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Vamorolone received FDA approval in 2023 for Duchenne muscular dystrophy in patients 2 years and older. It works similarly to standard steroids for maintaining muscle function but doesn't slow down growth like traditional steroids do. Talk to your child's doctor about whether vamorolone might be right for your family.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open muscular dystrophy trials
Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults
People with cerebral palsy (CP), muscular dystrophy (MD), spina bifida, or spinal cord injury often have muscle weakness, and problems moving their arms and legs. The NIH designed a new brace device, called an exoskeleton, that is worn on the legs and helps people walk. This study is investigating new ways the exoskeleton can be used in multiple settings while performing different walking or movement tasks, which we call ubiquitous use. For example, we will ask you to walk on a treadmill at different speeds, walk up and down a ramp, or walk through an obstacle course. Optionally, the exoskeletons may also use functional electrical stimulation (FES), a system that sends electrical pulses to the muscle to help it move the limb.
A Registered Cohort Study on FSHD1
The data to be collected is intended to help healthcare providers make important medical and financial decisions concerning FSHD1, through an enhanced understanding of the prevalence, progression and natural history of FSHD1.