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…
Pfizer has released the primary results from the Phase 3 clinical trial of tafamidis, an investigational drug being researched as a potential treatment for transthyretin amyloid cardiomyopathy. For more…
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…
Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…
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,…
In today's ever-changing - and downright weird - world, you'd be correct to say say (if not over-say) that technology is slowly killing us. But not in this case! Good…
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…
Major pharmaceutical company Pfizer recently announced the success of a new experimental product. The drug targets a disease called transthyretin cardiomyopathy, related to amyloidosis, a rare and dangerous disease that affects…
A story published on ESPN.com, and written by Jackie MacMullan, details NBA Hall of Fame inductee Nate "Tiny" Archibald's diagnosis of the rare disease amyloidosis. Amyloidosis occurs when a substance called amyloid builds…
According to an announcement from The Amyloidosis Research Consortium (ARC), the organization has launched version two of the My Amyloidosis Pathfinder (MAP) tool. The tool was developed as a way for…
Potentially good news arrived for patients with hereditary TTR amyloidosis, as the treatment drug inotersen received it's New Drug Application approval and will go into review by the U.S. Food…
Have you ever really considered everything your kidneys do? Can’t recall that Biology 101 class, huh? When you have a rare disease like amyloidosis, the danger of developing chronic kidney…
Exciting updates have come out of Alnylam Pharmaceuticals. They just released data from one of their highly anticipated trials for its leading rare disease drug. It looks like it did…
My grandma drank three cups of green tea every day. It didn’t matter if it was 98 degrees outside in the middle of August or the coldest day in December…
How often do you reflect upon all the genuine reasons to be grateful? What prompts you to shift your focus from the sense of self to others? Do you find…
PW Partner the Amyloidosis Foundation (AF) is hosting their annual amyloidosis research benefit and you won't want to miss it! The AF was founded by two amazing amyloidosis patients who strived…
"The Troubles" in Ireland/Northern Ireland go back to the 1700s, and they were aptly named. There's been a lot of fighting on both sides—those sides being the Irish and the English.…
If you think this summer is hot, last summer was on fire for amyloidosis advancements and support. Our partner, The Amyloidosis Foundation produces a seasonal newsletter for all those interested…
1. On Thursday, June 1st at 12 pm EST, 11 am central, there will be a seminar by the Amyloidosis Foundation to better understand this complex disease. It will be narrated by two physicians from…
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