Product Development for Rare Neurogenetic Disorders

May 8, 2019 (Noon–1 p.m. ET)


Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB), Best Pharmaceuticals for Children Act, NICHD




For this webinar, Mustafa Sahin, M.D., Ph.D., will provide an overview of the development of products used to treat individuals affected with rare neurogenetic disorders. He will review scientific, clinical, and regulatory considerations related to drug development.

Additionally, Dr. Sahin will:

  • Review basic principles of pediatric and rare disease product development, with emphasis in neurodevelopmental disorders.
  • Review recent advances in our understanding on rare neurodevelopmental disorders such as tuberous sclerosis, Rett syndrome, and Fragile X syndrome.
  • Discuss scientific, clinical, and regulatory hurdles to overcome to bring therapies to the clinic in these rare disorders.

Dr. Sahin is a professor of neurology at Boston Children’s Hospital and Harvard Medical School and has established and directs the Translational Neuroscience Center and the multidisciplinary tuberous sclerosis program at Boston Children’s Hospital.


George Giacoia, OPPTB, NICHD
Phone: 301–496–5589

Please note: Views expressed during NICHD-sponsored events do not necessarily reflect the opinions or the official positions of NICHD, NIH, or HHS.
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