https://www.statnews.com/2026/02/12/bridgebio-dwarfism-achondroplasia-infigratinib/
BridgeBio drug for genetic cause of dwarfism succeeds in key study
Feb 12, 2026 - The company's oral medicine for achondroplasia showed stronger results than a BioMarin therapy in a clinical trial.
genetic cause
https://www.genedx.com/blog/genetic-cause-cerebral-palsy
Evelyn’s Story: Discovering the Genetic Cause of Cerebral Palsy | GeneDx Blog
Mar 24, 2026 - Evelyn was initially diagnosed with cerebral palsy, but exome sequencing revealed CTNNB1 syndrome—changing her care and preventing serious complications.
genetic causecerebral palsystorydiscovering
https://cmt.blog/2020/06/09/new-discovery-of-a-genetic-cause-and-likely-cure-for-a-previously-unknown-subtype-of-cmt2/
A new discovery of a genetic cause (and likely cure) for a previously unknown subtype of CMT2 – CMT...
Published in Nature Genetics last month, the INC group's findings are a big deal, and there is a really cool story from the University of Miami's Miller School...
a new discoverygenetic cause
https://www.kth.se/en/om/nyheter/centrala-nyheter/cause-of-common-heart-valve-defect-revealed-in-genetic-study-1.1470676
Cause of common heart valve defect revealed in genetic study | KTH
heart valve
https://www.proqr.com/science/RNA-therapy
RNA therapy – Precision medicine targeting the underlying cause of genetic disease | ProQR...
Find out how RNA therapy works, how it differs from gene therapy and why we use it to treat genetic diseases that are currently untreatable.
rna therapyprecision medicinetargetingcausegenetic
https://deepmind.google/blog/a-catalogue-of-genetic-mutations-to-help-pinpoint-the-cause-of-diseases/
A catalogue of genetic mutations to help pinpoint the cause of diseases — Google DeepMind
genetic mutationsto helpthe causegoogle deepmindcatalogue