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
ASG Support Meeting
New HELIOS-B Study Update on Vutrisiran (AMVUTTRA®)
What this means for patients:
• New study results showed that people taking vutrisiran had better heart health results than those who did not receive the treatment.
• Heart scans showed less thickening and stiffness of the heart, which can help the heart pump more effectively.
• About 1 in 5 patients showed signs that amyloid buildup in the heart decreased, while no improvement was seen in people who received placebo.
• The treatment was linked to slower kidney damage, an important finding since ATTR can affect more than just the heart.
• For patients with serious kidney disease, vutrisiran was associated with a lower risk of death and major heart-related problems.
• Vutrisiran works by lowering the protein that causes amyloid buildup and is given as an injection four times a year.
These findings suggest vutrisiran may help slow ATTR-CM progression and support both heart and kidney health for some patients.
In the News
Phase 1 Study Update on Investigational Gene-Editing Treatment (nex-z)
What this means for patients:
• New early-stage study results showed that a one-time investigational treatment called nex-z sharply lowered the protein that causes ATTR for up to three years in people with ATTR-CM.
• Many patients showed stable or improved heart health over two years, even those with more advanced heart failure.
• Measures doctors use to track heart stress, heart damage, physical ability, and quality of life were stable or improved in most patients.
• Heart ultrasound results showed little to no worsening of heart structure at two years.
• A separate comparison analysis suggested lower death rates in patients who received nex-z compared to similar ATTR-CM patients who did not, though this was not from a direct trial comparison.
• Nex-z was generally well tolerated in this small study. Side effects were mostly infusion-related reactions and mild, temporary liver enzyme changes.
• Nex-z is a gene-editing therapy designed to turn off the gene that makes transthyretin (TTR). It is still experimental and not FDA-approved.
• Larger Phase 3 studies are planned but are currently on FDA clinical hold.
These early results suggest a one-time treatment approach may have long-lasting effects, but more research is needed to confirm safety and benefit.