Rare Disease Day: My hATTR Amyloidosis Story
Written by Diane Wilkie My hATTR story began in the mid-1960’s when my father, a healthy, strong, outdoorsy guy in his late 40’s started tripping, experiencing numbness, and having dizzy…
Written by Diane Wilkie My hATTR story began in the mid-1960’s when my father, a healthy, strong, outdoorsy guy in his late 40’s started tripping, experiencing numbness, and having dizzy…
Fredericksburg, Virginia resident Johnny Boatman is a former national guardsman who had been in excellent physical condition all his life. That was until 2022 when he felt a sharp pain…
According to a story on pharmaphorum.com, the UK's National Institute for Health and Care Excellence (NICE) has given a positive opinion for the drug vutrisiran (marketed as Amvuttra) as a…
A recent article in the Northern Kentucky Tribune highlights a rare condition that has been exposed by new technology. Cardiologists have found that many patients diagnosed with heart failure are…
CRISPR Cas9 gene editing is still looked upon as a new phenomenon in medicine. Yet it appears in the news almost every week. This week Neurology Today shines a light…
When it comes to advances in hereditary ATTR amyloidosis (hATTR amyloidosis), biopharmaceutical company Alnylam Pharmaceuticals, Inc. ("Alnylam") has been on top of its game. Alnylam helped develop Onpattro and is…
Cooking and eating together is a great way to build closer bonds and spend time with those we love. In Malaysia, it's a large part of the culture, as there…
Currently, there are no treatments available for patients with hereditary ATTR amyloidosis (hATTR); most available therapies are designed to aid with symptom management. However, biotechnology company and RNA-targeted therapeutics leader…
Over ten years ago two Nobel-Prize-winning biologists, Emmanuelle Charpentier and Jennifer Doudna, discovered a technology called CRISPR (clustered regularly interspaced short palindromic repeats). Bacteria contain genetic sequences repeated throughout…
According to a story from globenewswire.com, the clinical company Prothena Corporation plc recently presented encouraging results from its phase 1 clinical trial. This trial was evaluating its investigational product candidate…
According to a story from Newswise, the genome editing company Intellia Therapeutics has recently announced that the first patient has been dosed in its phase 1 trial, which will test…
According to a story from PR Newswire, Akcea Therapeutics, Inc. recently announced that the Portuguese Medicine Regulatory Authority (INFARMED) has announced its approval of reimbursement for inotersen (marketed as TEGSEDI®)…
According to a story from Guru Focus, the biopharmaceutical company Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. recently announced that the Ministry of Health of Spain has cleared…
A recent article in Bloomberg’s SFGate focused on the price of drugs. The average patient cannot afford the price of many drugs now on the market. Insurance companies have…
An article appeared recently in BioNews announcing the first-ever approval of an RNA-based therapy for use on the British National Health Service. The name of the drug is Patisiran…
According to a story from Nature, Dr. Carlos Heras-Palou was diagnosed with hATTR amyloidosis when he was just 39 years old. Initially, the devastating diagnosis was completely overwhelming, as the disease…
According to a story from BioPortfolio, Alexion Pharmaceuticals, Inc. and Eidos Therapeutics, Inc., have recently struck an agreement related to the development and marketing of an experimental treatment for hATTR…
According to a story from BioPortfolio, the drug company Alnylam Pharmaceuticals recently presented research findings related to hATTR amyloidosis, a rare disease. The study was presented at the Second European…
According to a story from Pharmafield, The UK's National Institute for Health and Care Excellence (NICE) has recently approved a first in class gene silencing therapy for coverage on the…
According to Globe Newswire, a pharmaceutical company, Akcea Therapeutics, recently announced their new genetic testing and counseling treatment program for those who may have hereditary ATTR amyloidosis (hATTR amyloidosis). The program,…