I have been involved in designing and directing clinical trials in pediatric neuromuscular disease for 30

years. I am currently Co-Director of the MDA Pediatric Care Center at Childrens Health (CH). Since 2015,

I have been Associate Director of the UTSW Wellstone Muscular Dystrophy Center funded by NIH, led by

Dr. Eric Olson. I am co-Chair of the Administrative Core for the Wellstone Center. I was PI and leader for

an NIH sponsored clinical trials group, AmSMART from 2000 through 2008, NIH funded, which established

for the first time reliability and validity for outcome measures in a young spinal muscular atrophy (SMA)

population. I was a member of the Outcomes Committee for the International Coordinating Committee for

SMA and Co-chair of the panel to develop Common Data Elements for Pediatric Neuromuscular Disease,

a project of the NINDS. I was a member of the panels that developed CDEs for congenital muscular

dystrophy and for SMA. I was an invited member of a Steering Committee to govern the project for

conducting IND enabling toxicology studies for scAAV9-SMN gene therapy. I have worked closely over

many years with several patient advocacy/volunteer groups including the Muscular Dystrophy Association,

Parent Project for DMD and Cure SMA. Furthermore, I served on the International Standard of Care

Committees for Congenital Muscular Dystrophies, SMA and the DMD Study Group. I serve on research

grant review committees as well as several planning committees and panels for patient groups and the

NIH. I am CoInvestigator for the UT Southwestern NeuroNEXT grant from NINDS which has been renewed

by NIH for the next 5 years (2018-2023). I have trained/mentored >20 pediatric neuromuscular fellows and

medical students, many of whom are currently in academic pediatric neurology positions. I hold 2-3

pediatric neuromuscular clinics per week, all of which are official MDA sponsored clinics. A large majority

of children diagnosed with DMD/SMA in north Texas are referred to my team either by their PCP, another

child neurologist or by parents who read about our program on the internet.