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To Arielle and her family, we appreciate all you have done in sharing your story with the  .When we visited you in North...
06/17/2026

To Arielle and her family, we appreciate all you have done in sharing your story with the .

When we visited you in North Carolina, we came hoping to better understand what life with ADH1 looks like. What we left with was so much more than that.

You reminded us why rare disease awareness matters in the very real, everyday lives of families like yours.

Listen to the full episode of Arielle’s journey: https://bit.ly/4tUabaO

From early childhood through the teen years, MyAchonJourney offers reliable information and age-specific resources to he...
06/16/2026

From early childhood through the teen years, MyAchonJourney offers reliable information and age-specific resources to help support families navigating .

Learn more at myachonjourney.com.

06/12/2026

🚨 Something big is coming to and we can't wait to show you.

We are about to unveil something that has never existed before: a first-of-its-kind mobile genetic testing van, arriving at The Endocrine Society’s ENDO 2026 to offer no-cost, on-site genetic testing through PreventionGenetics for ADH1 and hypoparathyroidism.

That means any attendee can walk up, complete a simple cheek swab, and take a real step toward a confirmed ADH1 diagnosis. At the conference.

Far too many people have gone years without a diagnosis. We built this genetic testing program because every one of those years matters.

This is just the start. After ENDO, the van hits the road on a U.S. West Coast tour to bring testing directly to communities.

Learn more: https://bit.ly/4aLT03T

06/10/2026

"They knew about the hypoparathyroidism and they never connected it to that."

Arielle told her son Sebastian's doctors for years: I have a calcium disorder. He has low calcium on his bloodwork. Could this be related to his seizures?

It wasn't until Sebastian was hospitalized and a specialist was called in that everything changed when he recommended genetic testing for .

This is what ADH1 looks like. This is what delayed diagnosis costs a family. And this is why we keep talking about it.

🎧 Full episode of the episode here: https://bit.ly/4tUabaO

This week at Patients as Partners Europe, we met Michael McGrath, founder of The Muscle Help Foundation and the first pe...
06/08/2026

This week at Patients as Partners Europe, we met Michael McGrath, founder of The Muscle Help Foundation and the first person living with LGMDR1 to lead expeditions to both the North and South Poles.

His story is a powerful reminder of something we never want to lose sight of: the people at the center of this work are extraordinary. They don't wait for the world to catch up. They move forward on their own terms.

We're grateful to share space with advocates like Michael. And we're committed to being impatient for patients.

What does it look like to live with a condition that affects your heart, your kidneys, your muscles, your brain — and st...
06/03/2026

What does it look like to live with a condition that affects your heart, your kidneys, your muscles, your brain — and still have doctors tell you it's not related?

That's the reality for Arielle, her son Sebastian, and her dad, who live with , a genetic form of hypoparathyroidism that prevents the body from properly regulating calcium levels.

In this episode of , Arielle opens up about her diagnosis journey, the health crises she's navigated, and what it finally took to get answers for her son after years of seizures, hospitalizations, and being dismissed.

It's a powerful story about what it means to live with a disease across generations and why awareness matters.

🎧 Listen to the full episode: https://bit.ly/4tUabaO

We were honored to support the Curelgmd2i Foundation European LGMD2I/R9 Patient & Family Conference in Kosor, Denmark!Pa...
06/02/2026

We were honored to support the Curelgmd2i Foundation European LGMD2I/R9 Patient & Family Conference in Kosor, Denmark!

Patients, families, clinicians, researchers, and advocates from over 20 countries came together, which was a powerful reminder of the global community rallying around this cause.

A highlight of the conference was the "Ask the Expert" Q&A, featuring three of the world's leading neuromuscular disease specialists moderated by Kelly Brazzo of the CureLGMD2i Foundation. People living with LGMD2I/R9 and families showed up with their questions and the experts delivered! Together, we move closer to answers.

On  , we stand with families managing life with  , a common genetic form of  .Emma has lived with ADH1 since infancy, ne...
06/01/2026

On , we stand with families managing life with , a common genetic form of .

Emma has lived with ADH1 since infancy, needing 12 doses of medication every day, through the night and at school.

When asked how she manages, Emma simply says, "You do what you got to do."

This kind of resilience deserves more than awareness. It deserves answers, earlier diagnosis, and treatments that match the weight of what families like hers carry every day.

Today, we raise awareness and stand with the community – because no one should face this alone.

  disrupts   regulation, impacting daily life in ways many don’t see.   is a common genetic form of hypoparathyroidism a...
05/31/2026

disrupts regulation, impacting daily life in ways many don’t see.

is a common genetic form of hypoparathyroidism and often goes undiagnosed and sometimes only uncovered after a family member receives genetic testing.

Earlier diagnosis means earlier answers. And answers can change lives.

On , we stand with the hypoparathyroidism community – not as bystanders, but as people who believe no one living with a genetic condition should have to wait years to understand what's happening in their own body.

The community is not alone, and we are just getting started.

The date is locked in for LGMD2I/R9. The FDA has accepted our NDA for BBP-418 for LGMD2I/R9 and set a PDUFA target actio...
05/27/2026

The date is locked in for LGMD2I/R9.

The FDA has accepted our NDA for BBP-418 for LGMD2I/R9 and set a PDUFA target action date of November 27, 2026, the date by which the FDA will complete its review.

LGMD2I/R9 is a progressive, life-shortening disease with no approved treatments. If BBP-418 is approved, it would be the first for LGMD2I/R9, and potentially for any form of .

For a community that has lived with no approved treatment options, timing is everything. We're racing toward potential approval and preparing for launch so that those with LGMD2I/R9 can be reached as quickly and safely as possible.

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