Rare diseases
Investigational molecules
Locations
105 Digital Drive, 94949-8703, United States
BioMarin Pharmaceutical Inc. focuses heavily on conditions affecting linear growth and bone development, including achondroplasia, hypochondroplasia, idiopathic short stature, Turner syndrome, short stature homeobox-containing gene deficiency, and Noonan syndrome. Its research also extends to pediatric growth impairment linked to inadequate response to human growth hormone.
The sponsor’s clinical activity in this area includes children, infants, and adolescents, with a strong emphasis on growth-related outcomes and long-term treatment experience.
Biomarin Pharmaceutical Inc. runs clinical trials in conditions such as Osteochondrodysplasia, Hypochondroplasia, Duchenne muscular dystrophy, Short stature, among others.
Clinical trials run by this sponsor – those enrolling participants first.
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