Rett syndrome is a progressive neurodevelopmental disorder that almost exclusively affects females. Only in rare cases are males affected. Infants with Rett syndrome generally develop normally for about 7 to 18 months after birth. At this point, they lose previously acquired skills (developmental regression) such as purposeful hand movements and the ability to communicate. Affected children often develop autistic-like behaviors, breathing irregularities, feeding and swallowing difficulties, growth retardation, and seizures. Most Rett syndrome cases are caused by identifiable mutations of the MECP2 gene on the X chromosome and can present with a wide range of disability ranging from mild to severe. ​

• Symptoms: seizures, respiratory issues, disrupted motor skills
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• 1 in 10,000 incidence rate

• Mutation in MECP2: X-linked genetic neurological disorder
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• 95% patients are girls

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PTP1B Inhibition: A Mechanism-Based Approach
for Treatment of Rett Syndrome

Krishnan et al (2015) J. Clin. Invest

• ​Rett syndrome is caused by mutations in MECP2 (Methyl CPG binding protein)
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• MECP2 normally suppresses PTP1B expression​
  
  
• ​PTP1B dephosphorylates critical developmental substrates including TRKB, the receptor for BDNF

• Following disruption of MECP2 function in Rett, PTP1B adopts a dominant role in suppressing signaling

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• Treatment with PTP1B inhibitors restores normal signaling

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DepYmed’s PTP1B Inhibitors are Safe, Bioavailable & Efficacious.

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