Rett syndrome (RTT) – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030

 Rett Syndrome (Also called: RTS, Cerebroatrophic Hyperammonemia) is a rare, non-inherited, X-linked dominant neurological disorder affecting brain development only in girls and is one of the most common causes of mental retardation in females. Girls with the syndrome show normal development during the first 6-18 months of life followed first by a period of stagnation and then by rapid regression in motor and language skills. The hallmark of Rett syndrome is the loss of purposeful hand use and its replacement with stereotyped hand-wringing. Screaming fits and inconsolable crying are common. With this syndrome, the girls typically survive into adulthood but are at risk of sudden unexplained death.

 

Etiology-

Rett Syndrome is caused by a mutation within the Methylcytosine-binding protein 2 (MECP2) gene. The MECP2 gene is located on the X chromosome. Between 90% and 95% of girls with Rett syndrome have a mutation in the MECP2 gene. Among families with a child affected by Rett syndrome the chance of having a second child with the syndrome is very low. Eight mutations in the MECP2 gene represent the most prevalent causes of Rett syndrome. The development and severity of Rett syndrome symptoms depend on the location and type of the mutation on the MECP2 gene

 

Epidemiology-

According to the published articles of Rett Syndrome Research Trust, the incidence of Rett Syndrome is estimated at 1 in 10,000 females; in the United States, approximately 30,000 children and women are affected by the condition. As per Chahil G and Bollu PC, et al, says that approximately 90% of reported cases of RTT inherit mutations of the methyl-CpG-binding protein 2 (MECP2) gene. The diagnosis of Rett syndrome is still based on clinical criteria and clinical presentation. Over 95% of females with classic Rett syndrome will have a mutation in the MECP2 gene

 

The competitive landscape of Rett syndrome (RTT) includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

KOLs insights of Rett syndrome (RTT) across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Rett syndrome (RTT) Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which Data Inputs with sourcing, Market Event, and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Read more: Rett syndrome (RTT) – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset                           Company                                 Stage

1          trofinetide         ACADIA Pharmaceuticals Inc.    Phase 3

2          NNZ-2566         Neuren Pharmaceuticals Limited            Phase 2

3          EPI-743            Edison Pharmaceuticals Inc       Phase 2

4          ANAVEX2-73    Anavex Life Sciences Corp.       Phase 2

5          fingolimod (FTY720)      Novartis            Phase 2

6          triheptanoin       Ultragenyx Pharmaceutical Inc   Phase 2