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…
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…
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…
Are you interested in learning about the latest trends and drug development in the rare disease field? Then you should check out a special Journalist Team Presentation from Global Data…
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…
The 2nd Annual Run For Your Life 5K/10K Patient Worthy is delighted to be a platinum sponsor of this exciting fundraiser for the Amyloidosis Foundation. This Halloween themed event will…
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…
The Amyloidosis Foundation is a non-profit organization that is committed to working and advocating for the amyloidosis patient community. The foundation is dedicated to supporting amyloidosis patients and their families…
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…
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…
The US Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug ALN-TTRsc02. Alnylam Pharmaceuticals Inc. is developing ALN-TTRsc02 as a possible treatment for transthyretin-mediated amyloidosis.…
UPDATE: Patisiran has been approved! Check out the story here. According to a story from Xconomy, the U.S. Food and Drug Administration could be close to approving a new drug…
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 a story from Rare Disease Report, the pharmaceutical company Alnylam Pharmaceuticals announced the results of Phase 3 trials for its experimental drug patisiran. The drug is in the…
Matt Millen played linebacker in the NFL for 12 seasons. He was a tough competitor and won 3 Super Bowl rings. For eight years, he was the General Manager of…
According to a story from news.vanderbilt.edu, Charlotte Haffner has been working hard to advocating on behalf of amyloidosis patients. She recently underwent a heart transplant and stem cell transplant in…
According to a story from femalefirst.co.uk, Jas Shaw of the electronic music project Simian Mobile Disco was recently diagnosed with AL amyloidosis. The group was planning a tour of the…
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