AFBS is proud to have launched several ongoing Nemaline Myopathy Natural History Studies to study disease development and pave the way for future clinical trials. This is a pivotal phase in our research endeavors: the data collected in these studies is fundamental to design and launch future human clinical trials and seek regulatory approval for treatments.
North American Prospective Natural History Study
We’re proud to announce that the North American Nemaline Myopathy Natural History Study is now enrolling! This observational study is critical and collects the “baseline” data needed to launch future human clinical trials. Simply put: we cannot get treatments approved without this study.
We are looking for:
- Children aged 0–18
- ACTA1 or NEB mutations
- Willing to participate in a 3-year observational study
5 Locations across North America
- West Coast: Stanford University (CA)
- South: UT Southwestern (TX) & St. Jude (TN)
- East Coast: Boston Children’s (MA)
- Canada/North: SickKids (Toronto, Canada)
Email Stanford Neuromuscular Research (neuromuscularresearch@stanford.edu) for more information and to join.
For more informationabout this study, visit: https://clinicaltrials.gov/study/NCT06774703.
Brazil Prospective Natural History Study
AFBS is also funding a natural history study in Brazil, helmed by Dr. Cristiane Moreno, which is already operational at the University of São Paulo.
- Children aged 0–18
- ACTA1 or NEB mutations
For more information on participating in the Brazil study, please email
cristianeammoreno@gmail.com.
Citizen Health Digital Retrospective Natural History Study
Your personal journey with NM holds immense scientific value. To make the Digital Retrospective Study a success, we need 100 US-based participants to launch this effort successfully. Your story is the missing piece researchers need to build a path toward effective treatments!
Open to US residents of ALL AGES living with ACTA1 or NEB-related NM. Both adult self-advocates and parents/caregivers are highly encouraged to sign up.
While our Prospective Study gathers clinical data over the next three years, the Retrospective Study works by securely gathering the medical records you already have. This allows researchers to access decades of valuable clinical data, deepening our understanding and providing the robust datasets needed to design and launch future clinical trials.
It only takes minutes and Citizen Health handles all the heavy lifting:
- Register Through the Form Below: Sign up in just a few minutes.
- Name Your Care Team: List the doctors and facilities where you received treatment.
- No Hassle: Grant permission for Citizen Health to collect and digitize your records. Your data is completely de-identified to protect your privacy.