Global Rett Syndrome Pipeline Landscape Report 2022: Comprehensive Insights About 15+ Companies and 15+ Pipeline Drugs –

Global Rett Syndrome Pipeline Landscape Report 2022: Comprehensive Insights About 15+ Companies and 15+ Pipeline Drugs –

Global Rett Syndrome Pipeline Landscape Report 2022: Comprehensive Insights About 15+ Companies and 15+ Pipeline Drugs –

DUBLIN–(BUSINESS WIRE)–The “Rett Syndrome – Pipeline Insight, 2022” clinical trials has been added to’s offering.

The publisher’s, “Rett Syndrome – Pipeline Insight, 2022,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Rett Syndrome pipeline landscape.

It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

The report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Rett Syndrome pipeline landscape is provided which includes the disease overview and Rett Syndrome treatment guidelines.

The assessment part of the report embraces, in depth Rett Syndrome commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Rett Syndrome collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Rett Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Rett Syndrome.

Rett Syndrome Emerging Drugs Chapters

This segment of the Rett Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Rett Syndrome Emerging Drugs

Trofinetide: Neuren Pharmaceuticals

Trofinetide (previously designated by Neuren as NNZ-2566) and NNZ-2591 are patented synthetic analogs of molecules which are derived from IGF-1 and occur naturally in the brain. IGF-1 is a growth factor stimulated by growth hormone. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical both for normal development and for responding to injury and disease.

ANAVEX2-73 (blarcamesine): Anavex Life Sciences

ANAVEX2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring cellular homeostasis. The drug is in phase II/III of clinical trial evaluation for the treatment of patients with Rett syndrome.

Rett Syndrome: Therapeutic Assessment

This segment of the report provides insights about the different Rett Syndrome drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Rett Syndrome

There are approx. 15+ key companies which are developing the therapies for Rett Syndrome. The companies which have their Rett Syndrome drug candidates in the most advanced stage, i.e. phase III include, Neuren Pharmaceuticals.

Rett Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Rett Syndrome therapeutic drugs key players involved in developing key drugs.

Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Rett Syndrome drugs?
  • How many Rett Syndrome drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Rett Syndrome?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Rett Syndrome therapeutics?
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Rett Syndrome and their status?
  • What are the key designations that have been granted to the emerging drugs?

Key Players

  • Neuren Pharmaceuticals
  • Anavex Life Sciences
  • Edison Pharmaceuticals
  • Novartis
  • Ultragenyx Pharmaceutical
  • GW Pharmaceuticals
  • Q-State Biosciences
  • AveXis
  • AMO Pharma
  • Neurolixis
  • Biohaven Pharmaceuticals
  • Echo Pharmaceuticals
  • Vyant Bio
  • Prilenia Therapeutics
  • StrideBio
  • Relmada Therapeutics

Key Products

  • Trofinetide
  • Blarcamesine
  • EPI-743
  • Fingolimod
  • REL 1017
  • Triheptanoin
  • Cannabidivarin
  • STRX 230
  • Research programme: Rett syndrome therapeutics
  • AVXS 201
  • AMO 04
  • Pridopidine
  • NLX 101
  • BHV 5000

For more information about this clinical trials report visit


Laura Wood, Senior Press Manager
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