First clinical trial of vosoritide for children with hypochondroplasia shows increased growth [Yahoo! Finance]
BioMarin Pharmaceutical Inc. (BMRN)
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Source: Yahoo! Finance
in children with hypochondroplasia, a genetic cause of short stature in children, according to researchers from Children's National Hospital. “This is the first medicine that has been developed to specifically target the pathway involved in hypochondroplasia,” says Andrew Dauber, M.D. , chief of Endocrinology at Children's National. “These findings will help inform future studies of vosoritide for addressing growth disorders.” This clinical trial funded by BioMarin is the first-of-its-kind to treat children with genetic short stature who do not have achondroplasia. The team found the annualized growth velocity increased by 2.26 standard deviation (SD) and the height standard deviation score (SDS) increased by 0.36 SD during the treatment period versus the observation period. Hypochondroplasia specific height SDS increased by 0.38 SD, according to the trial published in eClinicalMedicine The authors studied 24 children with hypochondroplasia. Half were female and 22 out of 24 h
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BMRN
Earnings
- 4/24/24 - Beat
BMRN
Sec Filings
- 4/26/24 - Form 10-Q
- 4/24/24 - Form 8-K
- 4/23/24 - Form 8-K
- BMRN's page on the SEC website