amyloidosis


Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose

Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose

June 24, 2022 7:30 AM EDT

CAMBRIDGE, Mass. and TARRYTOWN, N.Y., June 24, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced additional positive interim data from an ongoing Phase 1 study of their lead investigational in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. The data were presented in an oral presentation at the European Association for the Study of the Liver (EASL) International Liver Congress™ 2022, taking place June 22 – 26 in London.

Read article here


Alnylam Announces FDA Approval of AMVUTTRA™ (vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

Alnylam Announces FDA Approval of AMVUTTRA™ (vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

Jun 13, 2022

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jun. 13, 2022– Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) approved AMVUTTRA™ (vutrisiran), an RNAi therapeutic administered via subcutaneous injection once every three months (quarterly) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. hATTR amyloidosis is a rare, inherited, rapidly progressive, and fatal disease with debilitating polyneuropathy manifestations, for which there are few treatment options. The FDA approval is based on positive 9-month results from the HELIOS-A Phase 3 study, where AMVUTTRA significantly improved the signs and symptoms of polyneuropathy, with more than 50 percent of patients experiencing halting or reversal of their disease manifestations.

View the full release here: https://www.businesswire.com/news/home/20220603005487/en/




2022 “Light the Night for Amyloidosis” Awareness Campaign

2022 “Light the Night for Amyloidosis” Awareness Campaign

“Light the Night for Amyloidosis” is an awareness campaign created to draw attention to amyloidosis symptoms, diagnosis, treatment, and hopefully a cure! Since ‘Amyloidosis Awareness Month’ occurs in March, we are asking everyone to light up their porch/entryway with red bulbs for the month of March.

We have also contacted many US landmarks and businesses to do the same in March 2022. Below is a list of the approved requests, along with the date the lighting will occur.

View our growing list here.

We have red light bulbs available for purchase here!  Please join us in our “Light the Night for Amyloidosis” awareness campaign.


Amyloidosis Foundation Announces 2022 Research Grant Recipients

Amyloidosis Foundation Announces 2022 Research Grant Recipients

We are pleased to announce the funding of three new Junior Investigator Research Grants for 2022. The Amyloidosis Foundation Research Grants have been awarded to the following applicants:

K. H. Vincent Lau, MD of Boston University, Boston, MA. His research is titled “Evaluating Plasma Neurofilament Light Chain as an Early Biomarker for Polyneuropathy in V122I Hereditary Transthyretin Amyloidosis”.

Zainul S. Hasanali, MD, PhD of University of Pennsylvania, PA.  His study is “Targeting amyloidosis through study of calcium dependent endoplasmic reticulum resident protein folding chaperones and their effect on antibody production in plasma cells”.  

Taxiarchis Kourelis, MD of Mayo Clinic, Rochester, MN. His research is titled “Characterizing the role of the tissue immunome in the pathogenesis of renal AL amyloidosis”.

The Amyloidosis Foundation has awarded over $2 million to research since the inception of the grant program in 2005.

Click here for photos and to read more about their proposals.

Congratulations!


2021 Giving Tuesday

2021 Giving Tuesday

On November 30, 2021, people all around the world are coming together to tap into the power of human connection and strengthen communities and change our world. Will you be one of them? 

Amyloidosis Foundation will be participating in GivingTuesday and we need your help! 

By joining the GivingTuesday movement, you’re proving that in times of uncertainty, generosity can bring the whole world together. 

Here is how you can get ready to give:

  1. Mark your calendar 
  2. Give. On November 30, go to our donation link and give.
  3. Spread the word. Encourage your friends and family to join you in creating real impact on November 30 by sharing what our mission means to you and why you support our organization. Make sure to use hashtag #GivingTuesday and tag us so we can share!

Let’s rally together to build stronger communities. 

The Amyloidosis Foundation is a non-profit organization that supports research through our annual grant program. Our mission is to raise awareness of the disease within the medical community and the public, in addition to providing education, information, and support for patients and their families. 

In 2020, we raised over $15,000. This year our goal is $20,000 and we need your help!

Those who are interested in joining Amyloidosis Foundation’s #GivingTuesday initiative can click here.

Together we give. Share your #UNSELFIE. Use our template and share it on Facebook, Instagram, and Twitter.


Alnylam Reports Positive Topline 18-Month Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy

Alnylam Reports Positive Topline 18-Month Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Oct. 27, 2021– Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis, met all secondary endpoints measured at 18 months

Read the full press release here.



Spring Webinar Recording: Emerging treatment landscape for Cardiac Amyloidosis

Spring Webinar Recording: Emerging treatment landscape for Cardiac Amyloidosis

Listen to this informative webinar that was recorded on May 26, 2021on the Emerging treatment landscape for Cardiac Amyloidosis. The webinar was hosted by Mathew S. Maurer, MD a general internist and geriatric cardiologist with advanced training in heart failure and cardiac transplantation.  He is the Arnold and Arlene Goldstein Professor of Cardiology at Columbia University Irving Medical Center, Vagelos College of Physicians & Surgeons, where directs the Clinical Cardiovascular Research Laboratory for the Elderly (CCRLE). Dr. Maurer is a member of the Advanced Cardiac Care Center at New York Presbyterian Hospital – Columbia Campus. 

After the presentation, Dr. Maurer answered many interesting questions from patients, physicians and family members.

Please share this link with your family and friends to spread amyloidosis awareness.

LISTEN HERE