Rett Syndrome is an indication for drug development with over 50 pipeline drugs currently active. According to GlobalData, preregistered drugs for Rett Syndrome have a 100% likelihood of approval (LoA) indication benchmark. GlobalData’s report assesses how phase transition success rate (PTSR) and likelihood of approval (LoA) scores for pipeline drugs in Rett Syndrome compared to historical benchmarks. Buy the report here.
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Rett Syndrome overview
Rett syndrome is a neurodevelopmental disorder. This is caused by genetic mutation on MECP2 gene. Rett syndrome is described in four stages: Stage I or early onset, Stage II or rapid destruction, Stage III or the plateau, and Stage IV or the late motor deterioration stage. Symptoms include slowed growth, loss of normal movement and coordination, loss of communication abilities, breathing problems, pain, irregular heartbeat, and seizures. Risk factors include family history and gender. Treatment includes disease modifying drugs.
For a complete picture of PTSR and LoA scores for drugs in Rett Syndrome, buy the report here.
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