Dravet Syndrome Foundation feels strongly that research is our best hope for advancing treatments and eventually finding a cure for Dravet syndrome and associated epilepsies. Research is not just the responsibility of scientists and doctors. It is important that patients participate to whatever extent they are able.

Patients can participate in research in several ways, including: 1) sharing their information with scientists through patient registries so researchers can understand how Dravet syndrome affects individuals; and 2) volunteering for clinical studies or trials to help doctors understand how treatments affect patients. In rare diseases such as Dravet syndrome, it can be difficult for potential treatments to reach the required number of participants to be granted FDA approval. In the absence of large numbers, we must leverage the resources we do have, which include our strong patient community.

Clinical studies seek to answer questions such as:

  • Does this investigational drug work?
  • Does it work better than another medicine already available?
  • Does it cause any side effects?
  • Are there any other benefits that could improve patient quality of life?


The BUTTERFLY Study, conducted by Stoke Therapeutics, is collecting information about the
mental development, behavior, movement, communication skills, seizure frequency, and sleep
pattern of young people with Dravet Syndrome. The BUTTERFLY Study is an observational
study, which means that no investigational medications or other treatments will be provided.
Participants will continue to receive their usual care, and they will be observed by a team of
doctors and nurses over time.

For more information, please visit this link or email

 ZX008 Expanded Access Program
FAIRE studies now closed

Zogenix has closed screening in both of its FAIRE studies of ZX008 (fenfluramine) when used as adjunctive therapy for uncontrolled seizures in pediatric and young adult subjects with Dravet syndrome, and thanks patients and their families for their participation.

However, they are committed to developing and commercializing transformative therapies to improve the lives of patients and their families living with rare diseases. They believe the optimal approach to bring an approved therapy to the community is by creating clinical development programs designed with input from experts in the medical community, regulatory authorities and the patient community. Their goal is to quickly enroll eligible patients into clinical studies in order to demonstrate the safety and effectiveness of their investigational products, and to rapidly obtain the global regulatory approvals needed to make these therapies widely available to patients that need them around the world.

The primary preferred mechanism for access to investigational therapies is by participation in one of our clinical studies.  However, they recognize that all patients may not be able to take part in their clinical trials and face urgent medical need.  As a result they offer a limited expanded access program (also known as “compassionate use” or “early access”) for our investigational therapy ZX008.

When a request for expanded access to their investigational therapy is made outside of a clinical trial, they carefully consider each request based on the following criteria:

  • Scientific rationale for a possible therapeutic benefit
  • There are no satisfactory alternative therapies available to the patient
  • The patient is not eligible for a clinical study
  • Adequate supply of therapy
  • Local and regional regulations

Requests for access to experimental therapies must be made by a qualified and licensed physician by emailing Zogenix’s Medical Information Department at Zogenix looks to acknowledge such requests within five business days after receipt of the email. More information on their clinical trials and their expanded access program can be found at


ELEKTRA is a clinical trial for children with epileptic seizures associated with Dravet syndrome or Lennox-Gastaut syndrome. It is a Phase 2, international, multicenter, randomized, double-blind, placebo controlled study. It will test TAK-935 to find out if it can help lower the number of seizures experienced in these children. The ELEKTRA study is fully enrolling and is not recruiting new patients.

THANK YOU to the families who are participating in the ELEKTRA study!

More details at