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Alnylam has submitted a new drug application (NDA) to the FDA for vutrisiran, which could bring another treatment option to patients with ATTR amyloidosis. Vutrisiran received “Fast Track” Designation, which is a process designed to expedite the review of drugs to treat serious medical conditions and fill an unmet medical need, with the goal of getting new drugs to patients sooner. Both the NDA and fast track designation are encouraging signs for vutrisiran’s future in the TTR amyloidosis space. (more…)

The American Association of Heart Failure Nurses (AAHFN) has decided to meet virtually again this year due to the COVID-19 pandemic for their 17th annual meeting. Registration is now open and the program includes many hot topics in amyloidosis including the treatment landscape, COVID-19 implications, genetic testing in hATTR, and more.

In this webinar for patients and caregivers, Dr. Sandy Wong, Director of the Amyloidosis Program at the University of California San Francisco, will describe the process of stem cell transplantation for AL amyloidosis patients. Terry Fogaren, a nurse practitioner from Tufts University Medical Center, will follow with information on how to prepare for and best recover from the procedure. The presentations will be followed by a live Q&A session!

ARC's CEO, Isabelle Lousada, was told she had just weeks to live when she was diagnosed with AL Amyloidosis 25 years ago. A dedicated team of physicians at Boston Medical Center performed an experimental stem cell transplant, making Isabelle only the 14th person in the world to undergo this procedure for AL Amyloidosis. Isabelle made a remarkable recovery and has dedicated her life to advancing research and improving the lives of other amyloidosis patients. At just 31 years old, Isabelle,...

Register now for the Virtual Amyloidosis Masterclass sponsored by the Amyloidosis Research Consortium (ARC) and the Boston University School of Medicine (BUSM), to be held on April 9th, 2021. This 1-day live AMA PRA-accredited event is an exciting interactive educational event where attendees will learn about amyloidosis diagnosis, care and hear practical information about developing an amyloidosis center and more. (more…)