The fourth webinar in the series, “Amplifying the NM Community’s Voice”, focuses on the pathway to a Natural History Study for Nemaline Myopathy. Guest speakers included Leslie Hayes, MD, Pediatric Neurologist, Lindsay Alfano, PT, DPT, PCS and Research Partner, and Alan Beggs, PhD and Scientific Partner.

You can watch the recording of the webinar here:

Are you wondering how to get involved?

Stay connected through AFBS and the patient registry (CMDIR)

Provide input on the design of measurement tools

Participate in the natural history study when recruitment begins at a future date


  • Natural History Study: A standardized collection of information about an affected population to record what the disease course entails when no intervention is performed
  • Outcome Measures: A standardized method used within medical practice or research which is used to assess the effect, both positive and negative, of an intervention or treatment. Examples: grip strength, FVC, walk test, slurp test
  • Clinical Trial: A research study in which human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.

For more information on data literacy and medical research, watch this new NORD video.

If you are interested in submitting questions, opinions and/or ideas about outcomes measures, email