Dr. Charlotte Sumner talks about gene therapy for SMA

Dr. Charlotte Sumner talks about gene therapy for SMA

SMA Therapies

Good multidisciplinary care is recommended for people with SMA. This includes physical, occupational and respiratory therapies, as well as nutritional support. Increasing the amount of SMN protein produced by the body is now the main target of therapeutics development.

Current Therapies

People living with SMA often need continuous care from their families and from a variety of medical specialists for management of daily activities, nutrition, breathing, respiratory illnesses and to prepare for complications.

  • Dr. De Vivo describes the current treatments and therapies for SMA
  • Dr. De Vivo explains how therapies have dramatically increased the lifespan of SMA patients
  • Esther and Frank describe the therapies in place for their 2 year old daughter, Maeve

Potential New Treatments

There are several promising new treatments for SMA aimed at increasing amount of SMN protein. However it takes many years for researchers to translate research from “bench to bedside."

  • Dr. Sumner provides an overview of targets for SMA therapies
  • Dr. Sumner describes small molecule therapies for SMA
  • Dr. Sumner talks about gene therapy for SMA
  • Dr. Sumner describes how early intervention is crucial when treating SMA