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.
BMN 333 Versus Vosoritide in Children With Achondroplasia
FranceA study to evaluate the long-term safety and effectiveness of BMN 351 in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.
ItalyLong-Term Study of Vosoritide Safety and Effectiveness in Children with Hypochondroplasia Until Adult Height is Reached
FranceStudy of Vosoritide Safety and Effectiveness in Infants and Young Children (0 to 36 months) with Hypochondroplasia
FranceStudy on the Effects of Vosoritide for Children with Idiopathic Short Stature
FranceStudy of BMN 351 given by intravenous infusion to evaluate safety and tolerability in patients with Duchenne Muscular Dystrophy
ItalyStudy of Vosoritide for Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome Not Responding to Growth Hormone
FranceStudy on the Effectiveness and Safety of Vosoritide for Children with Hypochondroplasia
FranceStudy on the Long-Term Safety and Effectiveness of Vosoritide for Children with Achondroplasia
GermanyStudy on Long-Term Safety and Effects of Vosoritide in Children with Achondroplasia
FranceLong-Term Safety Study of Valoctocogene Roxaparvovec for Patients with Severe Hemophilia A
BelgiumStudy of pegvaliase injections compared to diet control in adolescents aged 12-17 with phenylketonuria (PKU)
GermanyIncrease visibility for your trials and help patients find your site through our platform.
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