According to a story from mdmag.com, a recent proof-of-concept style study suggests that a certain machine learning tool could be of benefit to patients living with the rare condition alkaptonuria.…
Christine Wright Christine Wright was an active, athletic, healthy mother of three. But in 2007 she started exhibiting strange symptoms that no one could give her an answer to. She…
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…
A big step forward for those with amyloidosis! The European Medicines Agency (EMA) granted Pfizer a positive recommendation for the approval of its treatment for amyloidosis, a rare disorder that…
Amyloidosis is characterized by stiffness and an inability to function in certain organs. Because of the danger this presents, it is important that doctors are able to monitor the…
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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…
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…
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