BridgeBio

ICYMI ✨ Behind this milestone is a community that has spent years turning hope into action.

Families living with LGMD2I/R9 have organized, advocated, educated, and pushed the field forward, helping make progress possible for those waiting for meaningful options.

FDA Priority Review accepted for BBP-418
Anticipated PDUFA date: November 27, 2026

Chronically low calcium ran in the family, but no one's first thought was "rare disease."

For Arielle (and later, her son Sebastian), years of seizures, specialists, and uncertainty would finally lead to genetic testing providing an answer: a diagnosis of autosomal dominant hypocalcemia type 1 ( ).

Arielle's strong advocacy for her son in the face of many challenges is all-too-common when navigating rare disease. Thank you to CNBC Cures for featuring Arielle's story in the latest installment of its ongoing coverage.

Read more here: https://cnb.cx/4vYHPOb

We just wrapped up two incredible days at the Wellstone Dystroglycanopathy Patient & Family Conference in Iowa City & and we couldn’t be more grateful.

People living with , families, researchers, and clinicians came together to deepen understanding of life with dystroglycanopathies, including LGMD2I/R9. Doug Sproule, M.D., our chief medical officer of BridgeBio Neuromuscular, shared updates on our research progress and practical steps the community can take today, including confirming genetic diagnoses and ensuring the new ICD-10 code (G71.036) is in medical records.

It was so wonderful to be in person with Curelgmd2i Foundation, LGMD Awareness Foundation, and LGMD2i Fund, and meet many others living with the condition who shared their experiences. The work you all do is extraordinary.

A heartfelt thank you to the University of Iowa Wellstone Muscular Dystrophy Specialized Research Center, Katherine Mathews, M.D. and Carrie Stephan for creating something truly special. We leave inspired and more motivated than ever to continue this work together. 💚

There are things you only learn by showing up.

Recently, our team had the privilege of attending advocacy conferences organized by three remarkable organizations: Associazione per l'Informazione e lo Studio della Acondroplasia (AISAC - Italy),
Association Burkinabè des Personnes de Petite Taille (APPT-France), and Bundesverband Kleinwüchsige Menschen und ihre Familien - BKMF e.V. (BKMF-Germany).

Together, these gatherings brought hundreds of individuals and families, advocates, and healthcare professionals from across the skeletal dysplasia community for connection, shared learning, and real conversation.

The most valuable moments weren't on the agenda. They happened in between – in the experiences shared, the questions asked, and the perspectives that deepen our understanding.

Those moments are why showing up matters.

AISAC, APPT, and BKMF: thank you for welcoming us. And to everyone who shared their perspectives with us: your voices help shape our work and strengthen our understanding.

This Father's Day, here's to the dads who help us through the hard days, celebrate the wins, and remind us that anything is possible.

To every father whose life has been touched by a genetic condition, we celebrate all that you are and all that you do.

Happy Father’s Day.

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 help support families navigating .

Learn more at myachonjourney.com.