Genetics

Onconova Therapeutics announced a multi-institutional research and clinical program to evaluate rigosertib in pediatric RASopathies, a rare group of rare syndromes.

Onconova Therapeutics (NASDAQ:ONTX) announced a multi-institutional research and clinical program to evaluate rigosertib in pediatric RASopathies, a rare group of rare syndromes.
As quoted in the press release:

The program will generate supportive non-clinical data and obtain early clinical experience in the pediatric setting with rigosertib, Onconova’s lead clinical candidate.
Rigosertib in currently being evaluated in a global Phase 3 trial (INSPIRE) for MDS patients after failure of therapy with Hypomethylating Agents (HMAs). A Phase 2 trial of oral Rigosertib combined with Azacitidine is aimed at patients with MDS and AML.
The RASopathies are developmental syndromes usually caused by mutations that alter the RAS subfamily and mitogen-activated protein kinases that control signal transduction. Together, the RASopathies represent a group of neurodevelopmental syndromes affecting more than 1 in 1000 individuals, according to RASopathies.Net.

Click here to read the full press release.

Source: globenewswire.com

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