New data in children under 5 years with achondroplasia showed treatment with VOXZOGO was associated with improvement in tibial bowing, a common cause of pain and impaired function Data from the largest retrospective, multi-year observational study of more than 600 children and adults with hypochondroplasia found significantly higher rates of...
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SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new research from studies of VOXZOGO® (vosoritide) in children with achondroplasia demonstrating positive impact on important health indicators, including arm span and bone density. The data will be presented, along with new data from studies of VOXZOGO in hypochondroplasia, at the...
SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced results from the pivotal Phase 3 ENERGY 3 trial evaluating BMN 401 in children aged 1-12 with ENPP1 deficiency, a rare, serious and progressive genetic condition. Deficiency in ENPP1 function results in a decrease in plasma inorganic pyrophosphate (PPi)...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) in late-onset Pompe disease (LOPD) were presented at the 31st Annual Congress of the World Muscle Society (WMS), in Hiroshima, Japan. These results from the PROPEL open-label extension...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ® (pegvaliase-pqpz) in adults with phenylketonuria (PKU). The latest findings further validate the long-term benefit and established...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced it has entered into an exclusive worldwide license agreement with GC Biopharma Corp. and Hanmi Pharm Co., Ltd. to develop and commercialize HM15421/GC1134A, an investigational long-acting enzyme replacement therapy for Fabry disease. HM15421/GC1134A is a human recombinant alpha-galactosidase A...
Sydney, Australia – The epigenetic ‘signature’ of a rare, hard-to-diagnose breast tumour has been found by scientists at the Garvan Institute of Medical Research. The discovery could lead to improved treatment guidelines and better outcomes for patients with this rare disease. Accounting for less than 1% of breast tumours, phyllodes...
REDWOOD CITY, Calif. — Biomea Fusion, Inc. (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing oral covalent small molecules to treat and improve the lives of patients with metabolic diseases and genetically defined cancers, today announced initial response data from the first two type 1 diabetes patients...
ROOTSTOWN, Ohio – As BioMendics prepares to share its progress during the 2026 J.P. Morgan Healthcare Conference and Biotech Showcase, the Ohio-based biotech is advancing what could become the first disease-modifying therapy for Epidermolysis Bullosa Simplex (EB Simplex). For families living with EB Simplex, each day can mean fragile blistering skin with constant...
TORONTO, Ontario — Biomind Labs Inc. (NEO: BMND) (OTC: BMNDF) (FSE: 3XI), a leading biotech company focused on developing the next generation of pharmaceuticals to treat patients suffering from neurological disorders by targeting the drivers of disease, today announced the successful conclusion of the Phase 2 clinical trial of its...