Wearing a revolutionary-era tricorn hat, doctor Mathew Maurer stood at a lectern in front of an audience of fellow cardiologists in Philadelphia, decrying the price of a new medication that…
In an article from the Orphanet Journal of Rare Diseases, a recent study provides an overview of the treatment of familial Mediterranean fever (FMF) that does not respond to the standard…
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 a story from home.cableone.net, the biopharmaceutical company Takeda Pharmaceutical Company Ltd. recently announced the latest updates to their phase 3 clinical trial which is testing the company's drug…
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 romania-insider.com, there are a total of 26 known cases of hereditary transthyretin mediated (HTTR) amyloidosis known in the country of Romania. However, 20 of these…
The first ever drug in the United States for treating transthyretin amyloid cardiomyopathy (ATTR-CM) has finally been approved by the FDA. It's amazing news for this patient community. But, you…
Transthyretin-Mediated Amyloidosis Cardiomyopathy The FDA has just approved the very first treatment for hereditary or wild-type transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM). ATTR-CM is a rare disease that occurs when amyloids, an…
Earlier this week, a press release from Alnylam Pharmaceuticals, Inc, announced the that the phase 3 study of their experimental acute hepatic porphyria drug Givosiran yielded positive results. According to the…
Familial Chylomicronemia Syndrome Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It's caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in…
About BridgeBio Pharma BridgeBio Pharmaceuticals was created in 2015 by veterans from the fields of academia and biotechnology. The company focuses on the development of new therapeutic treatments for genetic…
AL Amlyoidosis is a rare disease which causes a buildup of harmful proteins (amyloids) in organs such as the kidneys and the heart. This accumulation causes organ damage as well as…
Ionis Pharmaceuticals and Roche have been collaborating on a drug for Huntington's Disease which will treat the root cause of the condition. Currently, there are no such approved treatments, meaning…
Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…
According to a story from freepressseries.co.uk, 45-year-old Stephen Millichamp was recently diagnosed with AL amyloidosis. The news was dire, and Stephen was told that the disease would ultimately be fatal.…
For the last 3 decades, the only treatment that has been available for hereditary transthyretin-meditated amyloidosis was a liver transplant. The condition is caused by abnormal transthyretin proteins. These proteins…
According to a story from Pharma Voice, Dr. Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA) has recently released a statement in regards to efforts…
According to a story from Compelo, the medical technology company LivaNova is kicking off an international trial that will assess the use of autonomic regulation therapy as a treatment for…
According to a story from Reuters, a recent study found that mild exercise, such as walking, can significantly reduce the risk of heart failure in older, postmenopausal women. This large…
For any football fans here, Matt Millen needs no introduction. For the rest of us who are unfamiliar, Matt is a four-time Super Bowl-winning linebacker who has played for the Oakland…
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…
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