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Peptides are small chains of amino acids that act as messengers and building blocks throughout the body. Scientists and doctors can leverage peptides to interact with specific biological pathways in a targeted way. Take C-type natriuretic peptide (CNP), for example. CNP is key

“It’s been an incredible journey these past two decades as our research has not only helped enable advancements in achondroplasia, but opened the door for clinical trials in other skeletal conditions as well.” Dr. Ravi, Group Leader of Skeletal Biology at @mcri_for_kids

We announced data in #achondroplasia and #phenylketonuria today at #ACMGMtg25, underscoring the impact of our efforts to help people living with genetically defined conditions. Read our press release to learn more: bit.ly/4ibHrEZ

A biologic is a type of medicine that is made using a living organism, such as a cell. These complex molecules can perform specialized tasks in the body to target the underlying mechanisms of complex conditions. Our scientists are harnessing biologics to research innovative

Today we shared topline results from our pivotal trial in #PKU, highlighting our work to expand treatment options for adolescents living with this genetic condition. Read our press release to learn more: bit.ly/3G1XTt6

Meet PAH. A gene that encodes an essential protein for breaking down phenylalanine, an amino acid found in many foods. When PAH doesn’t work properly, phenylalanine can build up in the body, affecting brain function and development. This is the root cause of phenylketonuria

Meet IDUA. This gene tells cells how to make an enzyme called alpha-L-iduronidase, which helps to break down complex sugars known as glycosaminoglycans (GAGs). In mucopolysaccharidosis I (MPS I), a rare lysosomal storage disorder, changes in the IDUA gene can lead to an

Antisense oligonucleotides are small genetic sequences with big potential. By targeting specific sequences of messenger RNA (mRNA), they can fine-tune genetic instructions, helping regulate production of a specific protein in a targeted way. One powerful application is exon

We’re looking forward to sharing an update with investors on our Q1 2025 results tomorrow at 4:30 p.m. ET / 1:30 p.m. PT. Join our earnings call and webcast here: bit.ly/3GuSkDO $BMRN

Today we announced Q1 2025 results to the investor community, including 15% year-over-year total revenue growth. Read more in our press release: bit.ly/42YbUA7 $BMRN

We’re sharing new data in multiple skeletal conditions at the Joint Congress of ESPE and ESE and the PES Annual Meeting. These findings provide meaningful insights into #achondroplasia and build on our investigational research programs in #hypochondroplasia and #TurnerSyndrome.

“We have to know absolutely everything we can about this gene to help the most people we can.” BioMarin scientists are using innovative data science to learn more about variants in the FGFR3 gene and help improve the path to diagnosis for children with #hypochondroplasia, a

We’re excited to share that today we announced a definitive agreement to acquire @Inozyme, a clinical-stage biopharmaceutical company advancing an innovative enzyme replacement therapy for ENPP1 Deficiency, a condition with no currently approved treatments. ENPP1 Deficiency is

We presented new data at #PedsEndo2025 highlighting research on tibial bowing, a potentially painful complication affecting some children with #achondroplasia. Read more in our press release: bit.ly/3YHPsd8

Our global teams had a week full of inspiring connections and robust conversations with clinicians, researchers, advocates and patients in the pediatric endocrinology community at the Joint Congress of ESPE and ESE, and the PES Annual Meeting. We were grateful for this

Meet ARSB: a gene that produces arylsulfatase B, a key enzyme responsible for breaking down complex molecules called glycosaminoglycans (GAGs). If ARSB or arylsulfatase B aren’t functioning properly, certain types of GAGs can build up, leading to progressive damage in tissues and