Newron Pharmaceuticals is expanding the International Rett Syndrome Burden of Illness Survey to Europe and Australia in partnership with the worldwide Rett patient advocacy community, following strong participation from caregivers and medical professionals in the United States.
Launched last November in the U.S., the survey is being extended to the United Kingdom, Germany, Italy, and Australia to assess the physical, emotional, and economic impact of Rett syndrome on patient communities.
“We are very happy to be able to expand the outreach for our Burden of Illness survey on an international level,” Stefan Weber, CEO of Newron, said in a press release. “We are grateful that this study is strongly supported by so many Rett patient advocacy groups.”
Conducted under the banner “The Voice of Rett,” findings from the study will be used to improve programs and services for treating and managing the disorder.
Also, the survey will be the first to provide an estimate on the economic impact of a disease on specific countries, regions, communities, and individuals.
“Very little information concerning the true cost of Rett syndrome is available,” said Rachael Stevenson, executive director of Reverse Rett (UK). “As new advances in treatments for Rett are developed, the community urgently needs this information to support the case for funding.”
The survey is a chance to focus on the how Rett impacts not just the patient but the whole family, said Becky Jenner, CEO of Rett UK.
“With no dedicated treatment for any aspect of Rett syndrome, the study will help the whole community understand just why a treatment, even if it helps with just one symptom, is so very important,” she said.
Gabriele Keßler, first chairman of the Federal Association Rett Deutschland e.V., in Germany, said the survey is essential to “capture and publish individual caregiving situations for the very first time.”
A global Clinical Research Organization is in charge of conducting the survey through collaborations with Rett syndrome advocacy groups and experts including doctors and academics.
Final results from the survey will be published in academic journals to reach a wide audience.
Primary caregivers of at least one girl or woman with Rett syndrome being cared for at least 10 hours every week can participate in the survey. To participate, visit this website and click on the flag of your country on the upper right corner of the webpage.
In addition, Newron is conducting a Phase 2/3 clinical trial (NCT02790034) to test the safety, tolerability, and efficacy of sarizotan (10 to 20 mg) as a potential treatment for breathing problems or apneas in Rett patients.
The study, named STARS, is currently taking place in the U.S., United Kingdom, Italy, India, and Australia. Results are expected during the first half of 2020.
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