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…
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…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
