Ads
related to: clinical trials for amyloidosis- Heart Failure & ATTR
Recognize the signs & symptoms
Download the symptom tracker
- ATTR Overview
Learn the cause of ATTR
Know the different types of ATTR
- Resources
Download the patient brochure
Find support groups
- Care for Caregivers
Find tips for caring for ATTR
Manage the healthcare journey
- Heart Failure & ATTR
Search results
Results from the WOW.Com Content Network
The SAP-amyloid association has also been identified as a possible drug target for anti-amyloid therapy, with the recent development and first stage clinical trials of CPHPC for amyloidosis. [1] CPHPC has also been patented for possible treatment of Alzheimer's disease. [2]
Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis. [3] It is a transthyretin-directed antisense oligonucleotide. [3] It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca. [4] [5] [6] [7]
There was one trial that evaluated the benefits and side effects of tafamidis for the treatment of transthyretin amyloidosis with cardiomyopathy, in which patients were randomly assigned to receive either tafamidis (either 20 or 80 mg) or placebo for 30 months. [18]
Clinical trials: A phase I clinical trial (NCT02797847) was completed in January 2018, in which vutrisiran was evaluated to determine the safety, tolerability, pharmacokinetics, and pharmacodynamics in 80 healthy volunteers. [14] [18] It was a randomized, single blind, placebo- controlled study. [9]
Intellia has two in vivo programs in ongoing clinical trials. NTLA-2001 is an investigational CRISPR therapy candidate for the treatment for ATTR amyloidosis currently in Phase 1 studies. [3] NTLA-2002 is an investigational CRISPR therapy candidate for the treatment of hereditary angioedema (HAE) currently in Phase 1 / 2 studies. [4]
Inotersen blocks gene expression of both wild-type and mutant TTR, reducing amyloid precursor. Moderate-certainty evidence suggests that it mitigates worsening of peripheral neuropathy. Long-term efficacy and safety of inotersen use in people with mutant TTR-related amyloidosis is still be evaluated in a phase-III clinical trial as of 2021.
Acoramidis is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. [1] [4] [8] ATTR-CM is a rare and serious disease that affects the heart muscle. [4]
The FDA approved inotersen based on evidence from one clinical trial (Trial 1/NCT01737398) that included 172 participants with hereditary transthyretin-mediated amyloidosis. [6] The trial was conducted at 24 sites in Australia, Europe, South America, and the United States. [6] The benefits and side effects of inotersen were evaluated in one ...
Ads
related to: clinical trials for amyloidosis