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Press Release

Newron Pharmaceuticals Supports Rett Syndrome Awareness Month this October

Newron Pharmaceuticals
Posted on: 06 Oct 17

Milan, Italy & Morristown, N.J. – October 6, 2017 - 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 and peripheral nervous system, is pleased to support Rett Syndrome Awareness Month. October is dedicated to raising awareness for Rett syndrome through research initiatives, family empowerment and other advocacy events.


Stefan Weber, CEO of Newron, commented, “There are an estimated 36,000 girls living with Rett syndrome in the U.S. and the EU, with no approved treatments available. It is heartening to see the dedicated work advocacy groups such as have done to improve the lives of these girls. Newron is proud to support Rett syndrome awareness month and we look forward to further contributing to the Rett syndrome community.”


As Newron continues to advance its efforts for the Rett community, the Company is sponsoring the largest and most comprehensive qualitative study to examine the burden of Rett syndrome on individuals and their caregivers. A poster entitled “Burden of Disease in Rett Syndrome: A Qualitative Analysis” was presented at the ISPOR 22nd Annual International Meeting in May (International Society for Pharmacoeconomics and Outcomes Research) in the U.S. This poster presented 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.


Earlier this year the FDA approved the amendment of Newron’s ongoing Sarizotan Treatment of Apneas in Rett Syndrome (STARS) study to include Rett syndrome patients as young as six years of age. The potential benefits of sarizotan will now also be evaluated in these younger patients prior to the significant worsening that occurs as these patients age. STARS is a randomized, double blind, placebo-controlled study, designed to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome suffering from respiratory symptoms. Among the core symptoms of Rett, breathing disturbances may affect the whole body; they can have a marked effect on biochemistry, influence emotions, circulation and digestive function as well as musculoskeletal structures in the respiratory process.


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



About Newron Pharmaceuticals

Newron (SIX: NWRN) is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system. The Company is headquartered in Bresso near Milan, Italy. 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. Meiji Seika has the rights to develop and commercialize the compound in Japan and other key Asian territories. 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. For more information, please visit

Editor's Details

Mike Wood

Last updated on: 06/10/2017

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