What the trial was testing
The NEURO-TTRansform enrolled 168 patients with hereditary attr amyloidosis. The study was sponsored by Ionis Pharmaceuticals and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
82% drop in transthyretin protein levels and better nerve function.
JAMA · 2023 · NCT04136184
These findings — that in transthyretin protein and improved nerve function on monthly eplontersen — were published in the JAMA and represent the headline result of the study.
Researchers tracked outcomes across 168 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with hereditary attr amyloidosis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Eplontersen (Wainua) is FDA-approved and available now for hereditary ATTR amyloidosis with nerve disease. It is given as a monthly under-the-skin injection that can be done at home. Ask a neurologist about treatment for confirmed ATTR amyloidosis.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open hereditary attr amyloidosis trials
Phenotypic Manifestations of Hereditary ATTR Amyloidosis
This study focuses on hereditary transthyretin amyloidosis (ATTRv) with the Val50Met variant in a non endemic aerea
A Phase I/IIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of YOLT-201 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and participants with hereditary transthyretin amyloidosis with cardiomyopathy (ATTRv-CM).