🚐 Something is rolling into @theENDOsociety #ENDO2026 and it's never been done before. We're unveiling a first-of-its-kind mobile genetic testing van. On-site genetic testing through @PrevGenetics for ADH1 and hypoparathyroidism. One cheek swab. Zero cost. Come find us. Then watch us take it on the road. 🧬 Learn more: bit.ly/4aLT03T

Primary results from the CALIBRATE trial evaluating encaleret in ADH1 will be presented at @theENDOsociety’s #ENDO2026 on Saturday, June 13th at 2 PM CDT. Plus two posters on the genetics and patient experience of ADH1 on Monday, June 15.

Limb-girdle muscular dystrophy (LGMD) is a group of genetic neuromuscular disorders with many heterogeneous subtypes. Learn more about LGMD2I/R9 and the importance of identifying subtype at KnowLGMD.com.

"I'm really healthy all the time except for when I'm not. Then I'm living in the emergency room for several hours to days." Arielle lives with #ADH1. This is what that looks like. 🎧 Full episode of #OnRarePodcast here: bit.ly/4tUabaO

"No one's doing genetic disease research right now. I could call an academic after they publish and be the first company calling them."- Neil Kumar at #STATBreakthrough That's not a complaint. That's an opening. Three potential FDA approvals on the horizon. A pipeline built on contrarian bets. This is what building an independent biotech looks like.

AI can't compress a clinical trial. It can't generate the data we don't have yet. Our CEO Neil Kumar pushed back on the AI hype with @bloomberg at #MIGlobal with @MilkenInstitute – not because the tools aren't valuable, but because the years still take years.

Most drug programs start with a promising molecule. We start with the gene that's broken. Clearer biology = clearer signals. For rare disease communities still waiting on treatment, that difference is everything. 🎥 bit.ly/3RPZ9FD

For Arielle, growing up eating ice cream for breakfast wasn’t unusual, it was part of managing something no one fully understood. Her mom's reason: "Your calcium's low.” It wasn't until she was 16 – and years later for her son – that their family finally received a diagnosis: #ADH1. Her story is one you need to hear. 🎧 New episode of #OnRarePodcast here: bit.ly/4tUabaO

Honored to support the CureLGMD2i Foundation European LGMD2I/R9 Patient & Family Conference in Denmark! 20 countries came together for community, science, and hope. The "Ask the Expert" Q&A with Professor John Vissing (@uni_copenhagen), Dr. Katherine Mathews (@uiowa) & Professor Volker Straub (@UniofNewcastle), moderated by Kelly Brazzo, showed that when the community asks, experts can help to deliver. Thank you to CureLGMD2i for putting on event that moves critical dialogue forward!

On #WorldHypoparaDay, we stand with families managing life with #ADH1, a common genetic form of #hypoparathyroidism. Emma has lived with ADH1 since infancy. Twelve doses of medication a day through the night, at school, every day. How does she manage? "You do what you got to do." Her resilience deserves better. Earlier diagnosis. Real answers. Today, we raise awareness and stand with the community.

Too many people living with #hypoparathyroidism go years without a diagnosis. For #ADH1, a common genetic form, answers sometimes only come after a family member's genetic test points the way. Earlier diagnosis means earlier answers. And answers change lives. On #WorldHypoparaDay, the community is not alone. We're just getting started.

At the World Heart Summit and the Forum for Neglected Cardiovascular Diseases, the conversation kept coming back to the same truth: the science to address cardiovascular disease, including in the most neglected communities, already exists in many cases. What's missing is urgency and access. Thank you @worldheartfed for bringing the right people into the room and keeping the focus where it belongs.

Our Founder & CEO Neil Kumar on why rare disease is where we want to be. Full conversation with @Roivant CEO @gline on @BiotechTV's Off-Target Effects: biotechtv.com/post/off-targe…

The FDA accepted our NDA for BBP-418 for LGMD2I/R9 with PDUFA target action date: November 27, 2026. First-ever potential therapy for a disease that has never had one. $BBIO bridgebio.com/news

Some people with abnormal calcium levels may not realize their symptoms, like tingling, fatigue, or brain fog, are linked to a genetic hypoparathyroidism condition called #ADH1. A cheek swab for genetic testing takes seconds and can help families better understand their health and the health of future generations. Hear from families who came together with the @HypoPARAassoc to learn more with a simple cheek swab test: bit.ly/4bM7AcB

"No one's doing genetic disease research right now. I could call an academic after they publish and be the first company calling them."- Neil Kumar at #STATBreakthrough That's not a complaint. That's an opening. Three potential FDA approvals on the horizon. A pipeline built on contrarian bets. This is what building an independent biotech looks like.

New post-hoc exploratory data in @JCardFail examines serum TTR, cardiovascular mortality & hospitalization, and treatment with a TTR stabilizer in patients with ATTR-CM. Serum #TTR may serve as a clinically informative biomarker for ATTR-CM cardiovascular risk assessment following stabilizer initiation. Learn more: bit.ly/4wrDi7i

That's a wrap on #PES2026! 🙌 Grateful for many incredible moments: 3 booths on the floor, quality time with @HypoparthyroidismAssociation and other key voices in the community, and multiple skeletal dysplasia posters on site. See you next year!

Building a biotech to last is the rarest of things in this industry. Our CEO Neil Kumar will sit down with @Insmed CEO Will Lewis and @statnews senior biotech columnist @AdamFeuerstein on May 19 at the #STATBreakthrough Summit West in San Francisco. They'll talk about what it takes to stay independent, remain focused, and let nothing come between you and the patients. See you there.

This Women's Health Month, we're proud to partner with BlackDoctor and BridgeBio Pharma to spotlight a serious and often missed heart condition: ATTR-cardiac amyloidosis (ATTR-CM). New survey findings show there's a clear gap in awareness, especially among Black women. Many of them haven't heard of ATTR-CM or been screened. ATTR-CM is often underrecognized and underdiagnosed in women. And it is frequently mistaken for menopause or aging, which can delay diagnosis and early care. It's time to change that! If you have symptoms like unexplained fatigue, shortness of breath or swelling, ask your healthcare provider about ATTR-CM. Read the full announcement: prn.to/4txvpKM