On Wednesday, I will be speaking on real-world data at the International Angelman Syndrome Research Council's 2023 annual meeting and join members of the Angelman, CDKL5 and Rett syndrome communities to discuss advancements that have been made to bring therapies to patients living with these conditions. It is believed that these conditions share commonalities and that through collaboration, effective treatments can be found. #DrugDevelopment #Pharmaceuticals #RareDisease Pulse Infoframe Inc. Foundation for Angelman Syndrome Therapeutics
Femida Gwadry-Sridharโs Post
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๐ก๐ฒ๐๐ฟ๐ผ๐น๐ถ๐ ๐ถ๐ ๐๐ผ ๐ฝ๐ฟ๐ฒ๐๐ฒ๐ป๐ ๐ฝ๐ผ๐๐ถ๐๐ถ๐๐ฒ ๐ฟ๐ฒ๐๐๐น๐๐ ๐ผ๐ณ ๐ก๐๐ซ-๐ญ๐ญ๐ฎ ๐ฐ๐น๐ถ๐ป๐ถ๐ฐ๐ฎ๐น ๐ฝ๐ฟ๐ผ๐ผ๐ณ-๐ผ๐ณ-๐ฐ๐ผ๐ป๐ฐ๐ฒ๐ฝ๐ ๐ฎ๐ ๐๐ต๐ฒ ๐ช๐ผ๐ฟ๐น๐ฑ ๐ฃ๐ฎ๐ฟ๐ธ๐ถ๐ป๐๐ผ๐ปโ๐ ๐๐ผ๐ป๐ด๐ฟ๐ฒ๐๐ (๐ช๐ฃ๐). NLX-112, a first-in-kind, highly selective, serotonin 5-HT1A receptor agonist, completed a successful Phase 2A clinical trial for treatment of levodopa-induced dyskinesia in Parkinsonโs disease. The results from the trial, which met its primary and secondary endpoints, will be presented at the WPC in Barcelona. Presentations will take place as a ๐ฝ๐ผ๐๐๐ฒ๐ฟ ๐ผ๐ป ๐ช๐ฒ๐ฑ๐ป๐ฒ๐๐ฑ๐ฎ๐, ๐๐๐น๐ ๐ฑ (poster board # LBP38.31) and in an ๐ผ๐ฟ๐ฎ๐น ๐ฝ๐ฟ๐ฒ๐๐ฒ๐ป๐๐ฎ๐๐ถ๐ผ๐ป ๐ผ๐ป ๐๐ฟ๐ถ๐ฑ๐ฎ๐, ๐๐๐น๐ ๐ณ (morning Hot Topics session). In addition to clinical data, a poster on the neuroprotective effects of NLX-112 in a mouse model of PD will be presented on ๐ง๐ต๐๐ฟ๐๐ฑ๐ฎ๐, ๐๐๐น๐ ๐ฒ (poster board # P02.06). For further information: https://lnkd.in/e88qFW39 #parkinsonsdisease #drugdevelopment #serotonin #pharmaceutical #clinicaltrials #proofofconcept
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On 29 September, FDA announced the opportunity for a limited number of sponsors to participate in a new pilot program called Support for clinical Trials Advancing Rare disease Therapeutics that will offer additional meetings with the agency to sponsors of novel drugs that aim to treat rare diseases. In this article, we summarize the START pilot program, as well as other ongoing agency efforts to promote the development of drugs and biologics that treat rare diseases. #clinicaltrials #therapeutics #raredisease
