| Phase 4 |
| NCT07477691 |
Immune Modulation During Palynziq® Treatment in Adults (IMPALA) |
Phenylketonuria |
Recruiting |
| NCT06780332 |
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to… |
Phenylketonuria |
Active, Not Recruiting |
| Phase 3 |
| NCT07073014 |
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia |
Hypochondroplasia |
Enrolling by Invitation |
| NCT06455059 |
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia |
Hypochondroplasia |
Active, Not Recruiting |
| NCT03424018 |
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With… |
Achondroplasia |
Active, Not Recruiting |
| NCT06224907 |
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe… |
Hemophilia A |
Active, Not Recruiting |
| NCT05270837 |
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With… |
Phenylketonuria (PKU) |
Active, Not Recruiting |
| Phase 2/3 |
| NCT07441876 |
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With… |
Achondroplasia |
Recruiting |
| Phase 2 |
| NCT07126262 |
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36… |
Hypochondroplasia |
Recruiting |
| NCT06668805 |
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or… |
Noonan Syndrome |
Recruiting |
| NCT06382155 |
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature |
Idiopathic Short Stature |
Recruiting |
| NCT02724228 |
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children… |
Achondroplasia |
Active, Not Recruiting |
| NCT03989947 |
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With… |
Achondroplasia |
Active, Not Recruiting |
| NCT04554940 |
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia |
Achondroplasia |
Active, Not Recruiting |
| NCT07573631 |
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in… |
Duchenne Muscular Dystrophy (DMD) |
Enrolling by Invitation |
| Phase 1/2 |
| NCT05121376 |
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema |
Hereditary Angioedema |
Active, Not Recruiting |
| NCT04480567 |
AAV Gene Therapy Study for Subjects with PKU |
Phenylketonuria (PKU) |
Active, Not Recruiting |
| NCT06280209 |
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and… |
Duchenne Muscular Dystrophy |
Active, Not Recruiting |
| Phase 1 |
| NCT06738017 |
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants |
Alpha 1-Antitrypsin Deficiency |
Active, Not Recruiting |
| Not specified |
| NCT06212947 |
A Multicenter Multinational Observational Study of Children With Hypochondroplasia |
Hypochondroplasia |
Recruiting |
| NCT05579548 |
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to… |
Phenylketonuria, Maternal |
Recruiting |
| NCT06305234 |
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq… |
Phenylketonuria (PKU) |
Recruiting |
| NCT05813678 |
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) |
Phenylketonuria (PKU) |
Recruiting |
| NCT06309979 |
A Study to Assess Growth in Children With Idiopathic Short Stature |
Idiopathic Short Stature |
Recruiting |
| NCT06168201 |
VIrtual STudy in Achondroplasia for the US (VISTA) |
Achondroplasia |
Recruiting |