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Özge’s background is in research; she holds a MSc. in Molecular Genetics from the University of Leicester and a PhD. in Developmental Biology from the University of London. Özge worked as a bench ...
Patisiran was associated with a moderate improvement in cardiac symptoms in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a study found.
Tafamidis is linked to reduced all-cause mortality and a lower incidence of congestive heart failure exacerbations in ATTR-CM, a study found.
Using the new method, the researchers were able to successfully identify 15 cases of transthyretin mediated amyloid cardiomyopathy (ATTR-CM).
A multimodal approach combining vosoritide and limb lengthening led to gains in stature, gait, and emotional well-being in achondroplasia.
Conducting a RCT of the Accessercise app among adults with physical disabilities, including achondroplasia, is feasible and acceptable.
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