Rett Syndrome: Pipeline Analysis, Insight & forecast (2017-2025), H2 2017

• The report provides brief overview of Rett syndrome.
• The report highlights major unmet medical need in Rett syndrome diagnosis, patho-physiology, treatment & in prognosis.
• This report highlights FDA guidance for drug development in orphan disease & applicability of this guidance for Rett syndrome in terms of sample size, trial design & outcome measures (clinical endpoints).
• The report provides epidemiology data of Rett syndrome in US, Europe & Japan.
• The report maps pipeline molecule to unmet medical need of Rett syndrome.
• The report discusses residual unmet medical need which is not addressed by current pipeline drugs.
• The report provides a snapshot of the global therapeutic landscape of Rett syndrome.
• The report assesses Rett syndrome pipeline based on highest phase of development, type of sponsor, mechanism of action (MoA), route of administration (RoA), actionability, and molecule type.
• The report reviews Rett syndrome pipeline by companies and universities/research institutes based on information derived from company and industry-specific sources.
• The report covers pipeline products based on various stages of development for Rett syndrome ranging from discovery to pre-registration and undisclosed stages.
• The report encompasses benchmarking of all clinical products based on clinical trial data, highlights SWOT analysis of top five opportunities and top five mechanism of actions (MOAs).
• The report features descriptive drug profiles for the pipeline products which includes, product briefs, MoA description, rationale of development, FDA designation if any, expected launch year, clinical trial details with results if published, partnerships activity & current status.
• The report describes Rett syndrome market size in 2025 and sales forecast of top promising molecules from first year of their market launch to peak year.

Raptor Pharmaceuticals, Revive Therapeutics, ArmaGen, Inc, OPKOCURNA, Eloxx Pharmaceuticals, Mitochon Pharmaceuticals, Oryzon, SAGE Therapeutics, NeurotropeBioScience, Biohaven Pharmaceutical Holding Company Ltd, Orpheris, Inc., GW Pharmaceuticals, Anavex Life Sciences, Amorsa therapeutics, Neuragesis, Recursion Pharmaceuticals, Amicus Therapeutics, APTEEUS, AveXis, REGENXBIO, Neuren pharmaceuticals, Edison Pharmaceuticals, ORPHELIA Pharma, Novartis, Ultragenyx Pharmaceutical, Newron Pharmaceuticals

NNZ-2566 (Trofinetide), Dextromethorphan, rhIGF-1 (Mecasermin [rDNA]Injection), EPI-743, Desipramine chlorhydrate (ORP-002), Glatiramer Acetate, Fingolimod, Ketamine, Donepezil Hydrochloride, Triheptanoin (UX-007), Sarizotan (EMD 128130)