This 2-part first in human (FIH) study consists of an open-label, single ascending dose Part 1, which may identify the optimal biologically active dose (OBD)
Author: New Zealand Amyloidosis Patients Association (NZAPA)
NEURO-TTRansform Clinical Trials For AKCEA-TTR-LRx in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy A Phase 3 Global, Open-Label, Randomized Study
The NEURO-TTRansform Study
The NEURO-TTRansform Study is a Phase 3 clinical trial of an investigational antisense drug called AKCEA-TTR-LRx for people living with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). If you
Managing GI Issues In Patients With Amyloidosis & FODMAP
Nausea, bloating, indigestion, heartburn, cramps, diarrhoea, constipation, loss of appetite, etc. These are but a few of the issues that patients suffering from Amyloidosis face
To Inoculate or Not? Learn More About SARS-CoV-2 type Covid19 Vaccines
At the end of 2020 and now going into 2021, there have been ongoing discussions on the topic of Covid-19 and how we all can
How To Be Heard At The Doctors; Patient Voice Matters
The effectiveness of in-clinic, or virtually these days, visits to your doctor rests in your ability as a patient to make your doctor LISTEN and
Gene Editing Technology For Hereditary TTR Amyloidosis (hATTR) In New Zealand
What is hATTR Amyloidosis or FAP? Familial Amyloid Polyneuropathy (FAP) is a rare inherited disease caused by a specific mutation in your own DNA in a gene which codes for the Transthyretin protein (TTR). If you have this mutation, the TTR protein becomes sticky and forms clumps of amyloid within your small blood vessels and over many years this will damage your nerves (causing numbness and weakness) and heart (causing heart failure). Inheritance Traits These symptoms do not appear until a person is an adult. FAP is passed on between generations in an autosomal dominant pattern – which means if someone has the disease then half of his/her brothers and sisters will have the disease and half of his/her children will also develop the disease in the future. There are an estimated 50,000 patients with FAP worldwide and 100 in New Zealand. Present Treatment in NZ Up until now the only effective treatment for FAP has been liver transplantation, which replaces the liver which makes the abnormal TTR with one which does not. Unfortunately liver transplantation is very limited and to-date only 9 New Zealanders with FAP have been transplanted. CRISPR/Cas9
CRISPR/Cas9 Genome Editing Technology
HOW CRISPR/ Cas9 Works Video “CRISPR/Cas9 genome editing can make permanent, precisely targeted changes in patients’ chromosomes and repair the underlying genetic mutation, whereas more traditional
How My Pacemaker Contributed to Hypotension and Pedal Edema
“Pacemaker syndrome” is something which we chanced upon through a series of health mishaps that led to my collapsing in a chiropractor clinic one day
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