CAMBRIDGE, Mass.--(BUSINESS WIRE)--For many people living with a rare disease, such as hereditary ATTR (hATTR) amyloidosis, it can take years to be accurately diagnosed. Moreover, many may not realize that the symptoms they are experiencing can be tied to their family health history. As part of broader efforts to increase knowledge about hATTR amyloidosis, Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, has announced an initiative called Bridge the Gap to help patients and their families talk about the condition and recognize symptoms to gain a proper diagnosis.
Alnylam is partnering with a family that has lived with hATTR amyloidosis for generations to share their personal account in a book entitled, “Living a Rare Life.” This resource, along with other educational content, can be found at hATTRBridge.com.
“hATTR amyloidosis runs in my family and many of my relatives are living with the condition or have passed away from it,” said Angel, a caregiver for her family, several of whom carry one of the gene mutations that causes hATTR amyloidosis. “Early on, my family called the condition ‘the curse’ because no one knew what it really was and they experienced a wide variety of symptoms and misdiagnoses. I’m excited to be partnering with Alnylam on Bridge the Gap to share my family’s story and empower others to take action and seek support.”
hATTRBridge.com provides resources and information for those living with hATTR amyloidosis and their families, including a guide on how to initiate a conversation with relatives about health history, and tools to facilitate dialogue with a healthcare professional regarding genetic counseling and seeking a diagnosis.
hATTR amyloidosis is a rare, serious, progressive, life-threatening condition that is often passed down through generations. The condition is caused by a mutation in the transthyretin (TTR) gene that causes the TTR protein in the blood to misfold and form amyloid deposits throughout the body, resulting in a series of debilitating symptoms. hATTR amyloidosis is a multi-systemic disease, which impacts 50,000 people worldwide, and can lead to significant disability, decreased quality of life and a shortened average life expectancy.
“As one of the founders of a center that specializes in amyloidosis, I’ve witnessed firsthand the devastating impact a delayed diagnosis can mean for an individual’s quality of life,” said Sami L. Khella, M.D., Chief, Department of Neurology, Penn Presbyterian Medical Center and Professor of Clinical Neurology, University of Pennsylvania School of Medicine. “Through Bridge the Gap, we hope to raise awareness of hATTR amyloidosis to help those at risk of this complex condition identify symptoms and receive the proper medical attention they need to reach an earlier diagnosis.”
To learn more about hATTR amyloidosis and Bridge the Gap, visit hATTRBridge.com.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, and hepatic infectious diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform and deep pipeline of investigational medicines, including four product candidates that are in late-stage development. Looking forward, Alnylam will continue to execute on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam employs over 700 people in the U.S. and Europe and is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.