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Huntington’s disease (HD) is a neurodegenerative disease inherited in an autosomal dominant manner. It is caused by an expansion of cytosine, adenine, guanine (CAG) repeats within the huntingtin (HTT) gene, which is located on chromosome 4. This pathological expansion of CAG repeats results in the production of a mutant huntingtin protein with an abnormally long polyglutamine […]

Ingrid Scheffer, AES 2020 – Long Term Efficacy and Safety of Fenfluramine in Patients with Dravet Syndrome

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Published Online: Dec 11th 2020

We were delighted to talk to Ingrid Scheffer (The University of Melbourne, The Royal Children’s Hospital and Austin Health, Victoria, Australia) regarding the open-label extension study of fenfluramine in patients with Dravet syndrome (Clinical Trial Identifier: NCT02823145). The abstract ‘Efficacy and Tolerability of Adjunctive FINTEPLA (Fenfluramine Hydrochloride) in an Open-Label Extension Study of Dravet Syndrome Patients Treated for Up to 3 Years.’ (ABSTRACT NUMBER: 978) was presented at the virtual AES 2020, 4-8 December 2020.

Questions

  1. What are the advantages of fenfluramine compared with other anticonvulsants in patients with Dravet syndrome? (1:18)
  2. What clinical evidence led to the regulatory approval of fenfluramine in patients with Dravet syndrome? (1:58)
  3. What have the findings of the extension study taught us about the long term efficacy and safety of fenfluramine in patients with Dravet syndrome? (2:39)
  4. What safety issues should be considered when prescribing fenfluramine? (3:43)
  5. Which patients are likely to benefit most from fenfluramine and in whom is it contraindicated? (4:34)

Disclosures: Ingrid Scheffer has nothing to disclose in relation to this video.

Support: Interview and filming supported by Touch Medical Media.

Filmed as a highlight of AES 2020, 4-8 December, 2020 (Virtual).

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