BioMarin Pharmaceutical is an American biotechnology company headquartered in San Rafael, California. It focuses on therapies for rare and serious diseases and is publicly traded on Nasdaq as BMRN.
Ray Therapeutics received EMA PRIME designation for its RTx-015 optogenetic gene therapy for retinitis pigmentosa, following recent FDA RMAT status. The company also appointed Michael Murtagh as Chief Regulatory Officer to support global development.
Researchers have developed advanced platforms to study human neural networks, including 3D bioelectronics for brain organoids and scalable 2D neuron networks that reveal brain-like rhythms and drug responses.
BridgeBio Pharma reported positive Phase 3 results for oral infigratinib in children with achondroplasia, meeting primary and secondary endpoints with superior growth velocity and the first statistically significant improvement in body proportionality. The company plans regulatory submissions in the second half of 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07573631 |
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy |
ENROLLING_BY_INVITATION | PHASE2 |
| NCT07477691 |
Immune Modulation During Palynziq® Treatment in Adults (IMPALA) |
NOT_YET_RECRUITING | PHASE4 |
| NCT07473973 |
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency |
RECRUITING | PHASE3 |
| NCT07441876 |
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia |
RECRUITING | PHASE2/PHASE3 |
| NCT07126262 |
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months |
RECRUITING | PHASE2 |
| NCT07073014 |
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT06780332 |
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq |
ACTIVE_NOT_RECRUITING | PHASE4 |
| NCT06738017 |
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants |
ACTIVE_NOT_RECRUITING | PHASE1 |
| NCT06668805 |
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment |
RECRUITING | PHASE2 |
| NCT06455059 |
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia |
ACTIVE_NOT_RECRUITING | PHASE3 |
