MILAN & MORRISTOWN, N.J.--(BUSINESS WIRE)--Newron Pharmaceuticals S.p.A. (“Newron”) (SIX: NWRN), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system (CNS) and pain, today announced that a poster titled “Burden of Disease in Rett Syndrome: A Qualitative Analysis” will be presented at the ISPOR 22nd Annual International Meeting (ISPOR), taking place on May 20-24, 2017 at the Sheraton Boston Hotel and the John B. Hynes Veterans Memorial Convention Center in Boston, Massachusetts, USA.
The poster will be displayed on Tuesday, May 23, 8:30 a.m. - 2:00 p.m. ET, at ISPOR’s poster presentation session III, display location N19.
Rett syndrome is a severe neurodevelopmental disorder primarily affecting females, with an estimated prevalence ranging from one in 10,000. It is characterized by a loss of acquired fine and gross motor skills and the development of neurological, cognitive and autonomic dysfunction, which leads to loss of ability to conduct daily life activities, walk or communicate. This can put significant impact on individuals and their families, which can have a detrimental impact on quality of life.
This poster presents the results of a targeted literature search and preliminary findings from a qualitative interview study aimed at describing the burden of Rett syndrome on individuals and their families. Interviews are being conducted with caregivers and healthcare providers specializing in Rett syndrome to gain difference perspectives on the impact of Rett syndrome. Qualitative research is ideal for exploratory research like this as it provides an in-depth understanding of an individual’s thoughts, feelings and opinions.
Dr. Kate Williams, a Clinical Outcomes Assessment Consultant at PAREXEL International, a lead investigator on the study said: “We wanted to find out more about how the symptoms of Rett syndrome impact the lives of individuals and their caregivers. Therefore, we conducted this qualitative study to learn this information directly from the source.”
The literature review revealed that very few qualitative studies have been conducted to-date, and most of these focus on one specific symptom rather than the broader burden of illness on both individuals and caregivers. The analyses are ongoing.
The findings of the literature review and final qualitative analysis will inform the development of a conceptual model to describe the burden of Rett syndrome. This will provide a useful tool to inform the development of future questionnaires, including a planned large-scale international burden of disease survey which is due to be conducted in the U.S. and Europe at the beginning of next year.
About Rett Syndrome
Rett syndrome is a severe neurodevelopmental disorder primarily affecting females, with an estimated prevalence ranging from one in 10,000 females. There are no approved treatments available. Rett syndrome is characterized by a loss of acquired fine and gross motor skills and the development of neurological, cognitive and autonomic dysfunction, which leads to loss of ability to conduct daily life activities, walk or communicate. Rett syndrome also is associated with a reduced life expectancy. Approximately 25 percent of the deaths in patients with Rett syndrome are possibly related to multiple cardio-respiratory dysrhythmias that result from brain stem immaturity and autonomic failure. More than 95 percent of these patients have a random mutation in the MeCP2 gene. Episodes of apnea, hyperventilation and disordered breathing are found in approximately 70 percent of patients with Rett syndrome at some stage of their life. *For more information on Rett Syndrome, visit http://www.rettsyndrome.org.
About Newron Pharmaceuticals
Newron (SIX: NWRN) is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the Central Nervous System (CNS) and pain. The Company is headquartered in Bresso near Milan, Italy, with a subsidiary in Morristown, NJ, USA. Xadago® (safinamide) has received marketing authorization for the treatment of Parkinson’s disease in the European Union, Switzerland and the USA, and is commercialized by Newron’s partner Zambon. US WorldMeds holds the commercialization rights in the USA. In addition to Xadago® for Parkinson’s disease, Newron has a strong pipeline of promising treatments for rare disease patients at various stages of clinical development, including sarizotan for patients with Rett syndrome and ralfinamide for patients with specific rare pain indications. Newron is also developing Evenamide as the potential first add-on therapy for the treatment of patients with positive symptoms of schizophrenia. http://www.newron.com/en.
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