Edward Clinton Smith, MD

My clinical research interests focus on neuromuscular diseases. Neuromuscular diseases are a large group of disorders with various causes sharing one common feature: weakness.
One large group of neuromuscular diseases is caused by abnormalities in the nerves as they exit the brain stem and spinal cord and travel out to their respective muscles. These are called “neuropathies.” Common examples in this group include spinal muscular atrophy (SMA), Charcot-Marie-Tooth disease (CMT) and brachial plexus injuries. I am actively involved in clinical trials for SMA and follow over 40 SMA patients in my clinic. I also direct a clinic in collaboration with pediatric plastic surgery, orthopedics and occupational therapy to treat children with birth ("obstetric") brachial plexopathies.
Another large group of neuromuscular diseases, the “myopathies”, are caused by abnormalities in the muscle tissue. Some of the more common examples include Duchenne muscular dystrophy (DMD), myotonic dystrophy and facioscapulohumeral muscular dystrophy (FSHD). I am actively involved in clinical trials in this area and co-direct the Duke Children's Neuromuscular Program which was designated by Parent Project Muscular Dystrophy (PPMD) in June of 2015 as a "Certified Duchenne Care Center".
Less commonly, neuromuscular weakness is due to disorders of neuromuscular transmission. These are caused by abnormalities in the region where the nerve attaches to the muscle. Examples of disorders of neuromuscular transmission include myasthenia gravis and congenital myasthenic syndrome.
In addition to neuromuscular disorders, I have a strong interest in the management of cerebral palsy and spasticity. This can involve treatment with oral medications as well as EMG-guided chemodenervation ("Botox injections") and intrathecal baclofen therapy.
Since February 2015, we have offered multidisciplinary neuromuscular care for our patients in the Duke Children's Neuromuscular Program. This includes coordinated care from a pediatric neuromuscular specialist, pediatric pulmonologist, pediatric cardiologist, physical and occupational therapists, a nutritionist, a social worker and a medical equipment vendor - all in one location (Lenox Baker Children's Hospital). We also work closely with a pediatric endocrinologist, orthopedic surgeon, and a pediatric gastroenterologist, all with neuromuscular expertise. Genetic counseling services are also available.
Publications
Education and Training
- Child Neurology Residency Training, Pediatrics, Duke University, 2004 - 2007
- Residency Training, Pediatrics, University of Mississippi, 2002 - 2004
- M.D., University of Mississippi, 2002
Selected Grants and Awards
- NS Pharma NS-065 NCNP-01 DMD
- SHINE-CS11
- Multicenter OL PF 06939926 DMD Gene Therapy
- AAV2/8 LSPHGAA (Cohort II)
- Pfizer PF06252616 OL- EXTENSION in Boys with DMD
- An integrated and diverse genomic medicine program for undiagnosed diseases
- An integrated and diverse genomic medicine program for undiagnosed diseases
- Vamorolone-2B DMD
- A Long-term Observational Study Evaluating Eteplirsen in Patients with Duchenne Muscular Dystrophy under Conditions of Routine Clinical Practice
- Gene Therapy for Pompe Disease
- A Phase I Study of the Safety of AAV2/8 LSPhGAA in Late-onset Pompe Disease
- Cyto-CK-2127107 SMA
- Gene Therapy for Type 1 SMA
- SMA Care Center Registry
- 2019 PPMD CDCC Grant
- SOLID Duchenne UK DMD
- PPMD Research Liaison Grant
- NS Pharma - OL NS-065/NCNP-01 DMD
- VamoroloneVBP15-LTE
- Translational studies of GAA deficiency in bioengineered human muscle
- PPMD Research Liaison Grant
- Double Blind study to evaluate safety, efficacy, and pharma of PF 06252616 in boys with DMD
- A Comprehensive Newborn Screening Solution for Duchenne and Congenital Muscular Dystrophies
- Vamorolone 003
- Vamorolone 002
- CINRG PITT0112 Becker Muscular Dystrophy-A Natural History Study to Predict Efficacy of Exon Skipping
- Clinical Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic Trials
- Supplemental Funding for Phase 1/2 study of Clenbuterol for the Treatment of Pompe Disease
- OL Drisapersen in subjects with Duchenne Muscular Dystrophy
- Bloodwork_Clinical Outcomes Duchenne MD Therapeutic Trials-UCD0305
- Phase 1/2 Study of Clenbuterol for the Treatment of Pompe Disease
- CINRG CHAR0312 Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
- Clinical Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic Trials
- Clinical Trial Planning in Pompe Disease