Nexiguran ziclumeran (nex-z), an investigational CRISPR-Cas9 gene therapy for transthyretin amyloid cardiomyopathy (ATTR-CM), continues to demonstrate long-term efficacy in early-phase research, even as its phase III trials remain on hold…
vTransthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive heart condition driven by the buildup of abnormal transthyretin protein in the heart muscle and, at times, in nerves. For many living with…
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an infiltrative heart disease driven by protein instability. Normally a tetramer, transthyretin can dissociate into monomers that misfold, aggregate, and deposit as amyloid fibrils within…
The later part of 2023 will see decisions on drugs in four areas such as advanced melanoma, sickle cell disease, Alzheimer’s disease, and depression. BioSpace shines the spotlight on…
In a story from BioPharma Dive, a number of high-profile clinical trials are under way that could see conclusive results by the end of 2023. While some of these are…
The latest statistics on heart disease and its effect on Black communities are startling. According to an article in the Dallas Examiner, Black people are fifty to seventy percent more…
AstraZeneca has entered into two collaborative agreements which will improve research for two rare diseases: transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloidosis (ATTR). N1006 The first collaboration is with Neurimmune…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
Researchers have just announced that they have completed enrollment for a new Phase 3 trial for patients with transthyretin-mediated (ATTR) amyloidosis who have heart disease. This trial is examining a…
Pfizer Rare Disease is an approach taken by pharmaceutical giant Pfizer to develop treatments for rare and orphan diseases. In the past, the company has worked with the National Newspaper…
Pfizer has released the primary results from the Phase 3 clinical trial of tafamidis, an investigational drug being researched as a potential treatment for transthyretin amyloid cardiomyopathy. For more…
According to a story from Newswise, the results of a phase 3 clinical trial suggest that the medication tafamidis could substantially reduce the risk of hospitalization or death for patients…
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