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Latest News
BabyMERIT: U.S. Phase 3 Clinical Trial for Infants with RASopathy-Associated Hypertrophic CardiomyopathyBeth Stronach | Headlines | No CommentsThe Spark We've Been Waiting For We are ecstatic to share the announcement of BabyMERIT: the first U.S. phase 3 clinical trial to study the MEK inhibitor (Trametinib) in babies…
New International Research Survey from Boston Children's Hospital Recruiting participants for an anonymous online survey to explore the symptoms and impact of musculoskeletal pain in Noonan syndrome for adolescents and…
Suspension of BioMarin Clinical Trial of VOXZOGO® (vosoritide) in Noonan SyndromeBeth Stronach | Headlines | No CommentsBioMarin has made the decision to discontinue development of VOXZOGO® (vosoritide) for children with Noonan syndrome. Read the press release here.
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