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NLX-101 is a new drug being developed to treat breathing abnormalities in people with Rett Syndrome. It's possible that beyond breathing, the drug may also improve other core symptoms such as anxiety and movement disorders.
Categories
Beneficiaries
Situation
Many people with Rett Syndrome struggle daily with breathing irregularities, anxiety and movement disorders. There is currently no established effective treatment for any of these problems.
Solution
NLX-101 is a new drug under development by the pharmaceutical company, Neurolixis. NLX-101 has already been shown to normalise the irregular breathing patterns in Rett mice and has been given Orphan Drug status both in Europe and the US. Through this project, Reverse Rett will help Neurolixis, bring the new drug to the market, by funding the manufacturing and characterisation of clinical supplies of NLX-101 and supporting preparation for submission to the regulatory authorities.