@BioMarin Profile picture

BioMarin

@BioMarin

At BioMarin, we are committed to transforming lives through genetic discovery. Our community guidelines: https://t.co/w1kvdgjm98

Joined June 2009
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Natriuretic peptide receptor (NPR2) is a protein that plays a key role in bone biology. Activated by CNP, a master regulator of growth-related development, NPR2 promotes the growth of bone. At BioMarin, we’ve led research that has leveraged the NPR2 pathway for people living with…


We’re looking forward to the #TruePKU meeting next year, which will be the first opportunity for U.S. regulators to hear directly from the #PKU community in a public forum in more than a decade. We’re proud to support @NPKUA_Info's efforts to integrate the perspectives of people…

Announcing TruePKU! On May 8, 2025, join the global PKU community to share YOUR expertise on what it’s really like to live with PKU. Your lived experience with PKU is of great interest to FDA, life science companies, and others. Learn more at npkua.org/truepku #TruePKU #PKU

NPKUA_Info's tweet image. Announcing TruePKU! On May 8, 2025, join the global PKU community to share YOUR expertise on what it’s really like to live with PKU. Your lived experience with PKU is of great interest to FDA, life science companies, and others. Learn more at <a style="text-decoration: none;" rel="nofollow" target="_blank" href="https://t.co/u10ZUqo93T">npkua.org/truepku</a> #TruePKU #PKU


At BioMarin, we believe words matter and dehumanizing language is never okay. Please join us in standing with the dwarfism community by signing this petition advocating for the retirement of school mascots that promote stigma and harmful stereotypes. bit.ly/4geqQPv


Alpha-1 antitrypsin (AAT) is a protein made in the #liver that circulates throughout the body and protects tissues in the lungs from enzyme damage. It acts as an “off switch” that prevents enzymes such as neutrophil elastase from breaking down healthy tissue. People living with a…


This weekend, we’re presenting new real-world data that underscore the impact of our research for children with #achondroplasia at #ESPE2024. Read more here: bit.ly/3Of866x


Meet SERPINA1. This gene provides instructions for making the alpha-1 antitrypsin (AAT) protein, which forms in the liver and protects tissues in the lungs from enzyme damage. Changes in the SERPINA1 gene can reduce the body’s supply of AAT and cause a genetic condition called…


Today we announced third-quarter 2024 results, including 28% year-over-year total revenue growth. Investors can read more in our press release: bit.ly/4e4Vnhi $BMRN

BioMarin's tweet image. Today we announced third-quarter 2024 results, including 28% year-over-year total revenue growth. Investors can read more in our press release: <a style="text-decoration: none;" rel="nofollow" target="_blank" href="https://t.co/vodAZZcfHS">bit.ly/4e4Vnhi</a>

$BMRN

“The pull here was just too strong to ignore. BioMarin’s R&D is truly leveraging some of the greatest genetic science out there.” Since joining BioMarin at the end of September, Greg Friberg, Executive Vice President and Chief R&D Officer, has been spending time getting to know…

BioMarin's tweet image. “The pull here was just too strong to ignore. BioMarin’s R&amp;D is truly leveraging some of the greatest genetic science out there.” 

Since joining BioMarin at the end of September, Greg Friberg, Executive Vice President and Chief R&amp;D Officer, has been spending time getting to know…

We’re looking forward to sharing an update on our third-quarter 2024 results tomorrow at 4:30 p.m. ET / 1:30 p.m. PT. Investors can join our earnings call and webcast here: bit.ly/4fjPca7 $BMRN


Kathryn Lowell, our GVP, Global Government Affairs, has been recognized as one of the 2024 Most Influential Women in Bay Area Business by the @SFBusinessTimes Kathryn was honored both for her acumen as an industry-leading legislative strategist and her commitment to the…

BioMarin's tweet image. Kathryn Lowell, our GVP, Global Government Affairs, has been recognized as one of the 2024 Most Influential Women in Bay Area Business by the @SFBusinessTimes. Kathryn was honored both for her acumen as an industry-leading legislative strategist and her commitment to the…

Stories like Kainen's inspire us to continue working to help improve the experiences of people living with #Duchenne muscular dystrophy. As our team joins neuromuscular scientists and clinicians at the World Muscle Society Annual Congress in Prague, hear from Kainen’s family to…


An oligonucleotide is a molecule consisting of a short sequence of nucleotides, the building blocks of DNA and RNA. These molecules can alter the expression of genes. Oligonucleotides are one of several modalities our scientists are studying to develop breakthrough medicines for…


“The first day we brought her home, we knew we were going to treat her just like any other kid… Our hope for her future is that she conquers the world and does whatever she wants.” With her family’s support and encouragement, KK brings excitement, passion and fearlessness to…


We're pleased to join our partners @camp4tx in announcing a new research collaboration aimed at advancing novel therapeutics that increase protein levels by targeting regulatory RNA (regRNA) sequences, which are key elements controlling gene expression. Learn more:…


“Our lives could have been different without that early diagnosis… I’m hopeful that newborn screening continues to expand to cover more conditions and the goals of newborn screening will be fully realized.” Denise Queally still vividly remembers the moment in 2001 when she…

BioMarin's tweet image. “Our lives could have been different without that early diagnosis… I’m hopeful that newborn screening continues to expand to cover more conditions and the goals of newborn screening will be fully realized.” 

Denise Queally still vividly remembers the moment in 2001 when she…
BioMarin's tweet image. “Our lives could have been different without that early diagnosis… I’m hopeful that newborn screening continues to expand to cover more conditions and the goals of newborn screening will be fully realized.” 

Denise Queally still vividly remembers the moment in 2001 when she…
BioMarin's tweet image. “Our lives could have been different without that early diagnosis… I’m hopeful that newborn screening continues to expand to cover more conditions and the goals of newborn screening will be fully realized.” 

Denise Queally still vividly remembers the moment in 2001 when she…

We're excited to share new data at #ASBMR2024 that reinforce our pioneering research into leveraging CNP, one of the body’s own natural signals, and targeting the NPR2 pathway to impact growth in people with skeletal conditions. We’re committed to working to improve outcomes…


As we’ve gotten to know our 2024-25 RARE Scholars award recipients, we’ve been inspired by their stories and energized by their ambitions. Learn more about this year’s scholars – Allison Lowrie, Allyson Chan, Brian DuVal, Jiayi Yan and Will McClintock – and their academic…

BioMarin's tweet image. As we’ve gotten to know our 2024-25 RARE Scholars award recipients, we’ve been inspired by their stories and energized by their ambitions. Learn more about this year’s scholars – Allison Lowrie, Allyson Chan, Brian DuVal, Jiayi Yan and Will McClintock – and their academic…

“Kainen’s situation changed everything. It just gave you a broader perspective on how things could literally change at the blinking of an eye.” When Kainen received a diagnosis of Duchenne muscular dystrophy, his parents, Marcus and Cathy, recall how it affected their daily…


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