What is being talked about, taught, and shared right now in the transthyretin amyloidosis space. This page brings together key moments and opportunities so our community can stay informed and show up prepared.
What’s Happening in ATTR.
Upcoming Events
Community conversations, webinars, and programs happening across the transthyretin amyloidosis space. These events are hosted by trusted organizations and shared here for awareness.
Alnylam Patient Event
ARC Talks
Amvuttra Patient Education Event(s)
ARC V122I Webinar
Check out WomenHeart HeartTalks conversation on transthyretin amyloid cardiomyopathy (ATTR-CM), an often overlooked cause of heart failure that is frequently missed in women.
The discussion featured expert insight from Dr. Sarah Cuddy and powerful lived experience from Giselle Benmoin, a member of the Black community living with hereditary amyloidosis. Representation and awareness matter.
Grateful to WomenHeart for continuing to center women’s heart health through education and advocacy.
This video was created and produced by the Amyloidosis Research Consortium (ARC). All medical content and intellectual property belong to ARC.
#hATTRNextGen® integrates culture, faith, and community-centered dialogue specific to the V122I form of hereditary transthyretin amyloidosis. While much of the information on the #hATTRNextGen® platform may be applicable across the transthyretin amyloidosis space, ARC provides comprehensive resources spanning both AL and ATTR forms of amyloidosis. Visit ARC directly for broader research updates and patient support programs. www.arci.org
Closing Gaps in Cardiovascular Genetics: Heartgene Sciences Announces Launch
Heartgene Sciences has announced that it is officially live.
According to its public announcement, Heartgene was created to focus on heart-related genetics and to help close the gaps in diagnosis that affect patients and families living with inherited heart conditions.
What started as a program has now become a dedicated company, reflecting what they describe as a real and growing need.
Heartgene states that it provides clinical-grade genetic testing and genetic counseling at no cost to patients. The company also shares that it is committed to improving the experience over time, guided by input from clinicians, advocates, and families.
You can learn more directly from the company at:
https://heartgene.com/
A Note for the #hATTRNextGen® Community
Many in our community are impacted by hereditary transthyretin amyloidosis (hATTR), the V122I (also called p.V142I) form.
Heartgene’s announcement speaks broadly about inherited heart conditions. When it comes to hATTR and genetic testing, it is important to move thoughtfully.
Experts generally suggest that genetic testing may be considered for:
Adults who are having symptoms that raise concern
Adults with a known family history of the condition
Adult children, brothers, sisters, or parents of someone who has tested positive
For families with the V122I form, genetic testing is not usually recommended for children at this time, since this form most often develops later in adulthood. hATTR may show up at different ages even within the same family, so timing of testing should be guided by family history and conversation with a knowledgeable healthcare professional or genetic counselor.
In the News
Research Update: FDA Allows Gene-Editing Trial for ATTR Amyloidosis to Resume
In early March, the U.S. Food and Drug Administration (FDA) lifted a clinical hold on a major clinical trial studying a potential new treatment for transthyretin amyloidosis (ATTR). The therapy, called nexiguran ziclumeran (nex-z), is being developed by Intellia Therapeutics and uses CRISPR gene-editing technology.
The treatment is designed to turn off the gene that produces the transthyretin (TTR) protein. In hereditary amyloidosis, mutations in this gene cause the protein to misfold and form amyloid deposits that can damage the heart or nerves. The trial had been paused in 2025 after a safety event involving severe liver problems in one patient. After reviewing additional safety information and updated monitoring plans, the FDA allowed the study to move forward again with enhanced liver monitoring and stricter patient eligibility criteria.
The Phase 3 trial, known as MAGNITUDE, will study the therapy in patients with ATTR cardiomyopathy and could enroll around 1,200 participants worldwide. Researchers hope that gene-editing therapies like this could one day offer a single-dose treatment that reduces or stops production of the TTR protein, potentially slowing or preventing amyloid buildup.
While this treatment is still experimental and undergoing clinical trials, the FDA decision allows researchers to continue studying whether it could become a future option for people living with ATTR amyloidosis. Read the full article:
Read more about the FDA decision and Intellia trial