- Develop an approach to neuromuscular symptoms and signs that identifies children in need of expedited neuromuscular assessment, including the impact of recent developments such as genetically targeted therapies and expanded newborn screening on improved outcomes with timely and accurate diagnosis.
- Describe the psychosocial impact of neuromuscular disorders and strategies for supporting optimal function throughout childhood and preparing for adulthood.
- Outline the novel therapeutic approaches actively under study and on the near horizon in pediatric neuromuscular research clinical trials.
- Maximize the efficiency and efficacy of an interdisciplinary care approach in the context of introducing complex novel therapies to multisystemic disorders such as neuromuscular disease.
- 6:30 p.m. Welcome and Introduction
- 6:35 p.m. “Who needs a Neuromuscular referral? The importance of making an early diagnosis”, Elizabeth Kichula, MD and Brianna Gross, MS, CGC
- 7:00 p.m. “Maximizing school, home and social function in neuromuscular disorders and preparing for adulthood”, Andrea Paterno, RN and Sarah Stoney MSW, LSW
- 7:25 p.m. “Neuromuscular Clinical trial Landscape”, John Brandsema, MD (No CME)
- 7:50 p.m. “Integrating complex therapies into the clinic and optimization of interdisciplinary care”, Susan Matesanz, MD, Sabrina Yum, MD
- 8:15 p.m. Q&A; Adjourn
- John Brandsema, MD, attending neurologist with the Division of Neurology at Children's Hospital of Philadelphia.
- Brianna N. Gross, MS, CGC, genetic counselor with the Division of Neurology at Children's Hospital of Philadelphia.
- Elizabeth Kichula, MD, PhD, attending neurologist with the Division of Neurology at Children's Hospital of Philadelphia.
- Susan Matesanz, MD, attending neurologist with the Division of Neurology at Children's Hospital of Philadelphia.
- Andrea Paterno, BSN, RN, nurse with the Neuromuscular Program at Children's Hospital of Philadelphia.
- Sarah Stoney, MSW, LSW, Social Worker in the Pediatric Multiple Sclerosis Clinic, Neuromuscular Program, and Leukodystrophy Center at Children's Hospital of Philadelphia.
- Sabrina Yum, MD, attending neurologist with the Division of Neurology at Children's Hospital of Philadelphia.
ACCME Accreditation Statement: Children’s Hospital of Philadelphia is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
AMA Credit Designation Statement: Children's Hospital of Philadelphia designates this live activity for a maximum of 1.75 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Disclosure Statements: Brianna Gross, MS, CGC (Speaker), Elizabeth Kichula, MD, PhD (Speaker), Sarah Stoney, MSW, LSW (Speaker), Katie Lockwood, MD (Course Director) and Tina Mannices (CME Planner) and others as appropriate have no relevant financial relationship with an ineligible company to disclose.
Susan Matesanz, MD (Speaker) has a financial relationship that has been mitigated though the CHOP CE Peer-Review Mitigation Process.
Dr. John Brandsema (Speaker) has relationships with ineligible companies that cannot be mitigated. No AMA PRA Category 1 Credit™ will be awarded for his lecture.
Related specialties: neurology, neuroscience center
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