Bringing CNS Members Together to Make Children’s Lives Better


Latest News from CHOP

Dear Colleague,
We are pleased to announce that Children’s Hospital of Philadelphia (CHOP) was ranked the No. 4 pediatric neurology program in the country by U.S. News and World Report for 2021-22. This honor recognizes our excellence and expertise with regards to care and treatment, our commitment to patient safety, our emphasis on family-centered care, and our groundbreaking research. As one of the top pediatric neurology programs in the nation, our team is committed to sharing our advancements to further our collective effort to optimize care of patients and better understand neurologic disorders.

We hope you find the latest updates from our program, shown below, valuable and, as always, welcome your feedback. For questions or concerns, contact Marissa Malcolm, Administrative Director of CHOP’s Division of Neurology, at

If you are looking to consult with a CHOP pediatric neurologist or refer a patient for a second opinion, email

In This Issue:

  • Introduction to Our New Pediatric Neuroscience Seminar Series
  • Outcomes in Children with SMA After Gene Replacement Therapy
  • Clinic Helps Children with BPAN and WDR45-related Disorders
  • Team Member Leads Pediatric Movement Disorders Program

Introducing Neuro Connections: A Pediatric Neuroscience Seminar Series

In this new and free virtual education series, experts from our Divisions of Neurology and Neurosurgery lead live and recorded webinars discussing the latest advancements in neuroscience care and research.

Learn more about our upcoming webinars.

Outcomes in Children with SMA After Gene Replacement Therapy

William Jr. was diagnosed with spinal muscular atrophy (SMA) a little after he turned 1. Fortunately, just a few months later, a one-time gene replacement therapy was approved by the FDA for treatment of children under age 2 with genetically confirmed SMA. Within a month of receiving the gene therapy, William improved from being unable to roll over or lift his arms to being able to lift his arms over his head and bear weight on his legs. He has continued to gain strength and achieve milestones like crawling, standing, and taking steps. More than 20 patients have received gene replacement therapy at CHOP. All can now walk independently and are achieving normal developmental milestones.

Read more about William’s experience with gene replacement therapy.

Specialized Clinic Helps Children with BPAN and WDR45-related Disorders

Families from across the country are accessing care at our new Clinic for BPAN and WDR45-related Disorders. Members of our team are internationally recognized specialists in the unique management of these children. During visits to the clinic, families consult with neurologists and genetic counselors with specialty training and experience in the diagnosis and management of neurogenetic conditions, as well as occupational and physical therapists. All patients are offered the opportunity to participate in research studies assessing the natural history of these disorders and collecting biospecimens to further our understanding of BPAN and WDR45-related disorders.

Learn more about how the Clinic helps families at

New Provider Tapped to Lead Pediatric Movement Disorders Program

We are pleased to announce that our new Pediatric Movement Disorders Program will be led by Nivedita Thakur, MD. She received her medical degree from McGovern Medical School UT Health, where she also completed a residency in pediatrics, a fellowship in child neurology, a fellowship in pediatric movement disorders and neurodegenerative diseases, and a health educator fellowship. Dr. Thakur previously served as an attending physician at Memorial Herman Hospital and Shriners Orthopaedic Hospital, both in Houston, Texas. Dr. Thakur enjoys patient care in both outpatient and inpatient settings, she is enthusiastic about teaching the next generation of pediatric neurologists, and she is committed to innovative clinical research in pediatric movement disorders.

Stay tuned for more info about this new program.

PLEASE NOTE: This email was sent to CNS members with the approval and cooperation of the Child Neurology Society. A fee was paid to the Child Neurology Society for providing this email service. Official notice is hereby given that the CNS does not officially endorse the product, service, research/study, position, event or company/institution referenced, nor is any implication of endorsement intended by the contracting party. The CNS never releases member email addresses. Members may decide individually to provide their own email to third parties but are strictly prohibited from providing any member emails other than their own for any reason.

Click to review CNS Legal Privacy Policies.