Reflections on V122I, Genetic Risk, and the Questions Black Families Are Still Asking Editor's Note: This article was written and submitted to us by Erin Poyant of #hattrnextgen®. "When you…
In a news release from biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. ("Alnylam"), the company shared that positive data was available from the Phase 3 APOLLO-B clinical trial.…
A recent article on Yahoo Finance, reports that Alnylam Pharmaceutical Inc.’s RNAi therapeutic, Amvuttra, has been approved by the FDA to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. Amvuttra (Vutrisiran)…
According to a recent article, researchers have created a new screening tool that will aid in discovering if patients with chronic idiopathic axonal polyneuropathy have the TRR mutation. Hereditary Transthyretin…
According to a recent article from Donegal Daily, patients with hereditary amyloidosis in Donegal, Ireland created a film on the condition that has been plaguing parts of Ireland for ages.…
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…
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…
On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…
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…
According to a story from Biospace, the RNAi therapeutics company Alnylam Pharmaceuticals Inc. recently announced that its experimental product candidate vutrisiran has earned Fast Track designation from the US Food…
According to a press release from FirstWord Pharma, Gen and Alnylam Pharmaceuticals have signed an exclusive distribution deal in Turkey for RNAi therapeutic ONPATTRO. This therapy drug will treat…
As originally reported in Pfizer, the European Commission has just approved the first treatment option ever available to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). The medication, VYNDAQEL, taken orally once daily,…
Amyloidosis is a rare disease that has gone without awareness for a long time. This lack of awareness led to slow developments in treatment, issues with obtaining a diagnosis, and…
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Pfizer issued a statement this week through its Media Relations that its drug VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy received positive opinion by the European Medicines Agency’s (EMA) Committee for…
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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, 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 Wapakoneta Daily News, Greg was devastated when he learned that his brother was diagnosed with hereditary transthyretin mediated (hATTR) amyloidosis. Ultimately, the disease would take…
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It has been nine years since Pfizer acquired the drugs vyndagel and vyndamax. According to a report in Biospace, based on the FDA’s recent approval of the two drugs to treat transthyretin-mediated…
According to a story from the Irish Examiner, treatment with the recently approved drug patisiran helped a British surgeon named Carlos Heras-Palou save both his hands and his career. Patisiran…
According to a story from Business Wire, the therapeutics company Alnylam Pharmaceuticals recently announced that the company's drug patisiran has gained approval from the European Commission. Patisiran was approved for…
According to a story from bioportfolio.com, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval…
The final results from a study of the experimental drug tegsediTM (inotersen) for the treatment of patients with hereditary ATTR amyloidosis with polyneuropathy have been published. The original article can be…
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,…
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