Newsroom

Information on amyloidosis is provided for informational purposes only and is not intended to be medical advice. Information concerning medical care or the suitability or use of any medication should be discussed with a medical doctor. Any information on specific treatment protocols in not an endorsement of said product.


3RD European ATTR Amyloidosis Meeting for Patients and Doctors

3RD European ATTR Amyloidosis Meeting for Patients and Doctors

The 3rd European ATTR Amyloidosis meeting will be hosted 100% virtually on September 6-8, 2021. There will be 2 Channels, one dedicated to doctors and one dedicated to patients, across 3 days delivering a packed and high-quality scientific programme including cutting-edge lectures by international leaders in their field, sessions in collaboration with partnering companies, poster sessions, and much more. It’s the perfect place to keep you updated about ATTR amyloidosis!

Registration is open! You can now book your virtual seat!
 Register now

Submission is open! Be part of the programme by submitting an abstract
before July 18, 2021.
 Submit here

Jonathan Wall receives $1.79 million to develop amyloidosis treatment

Jonathan Wall receives $1.79 million to develop amyloidosis treatment

By BIOENGINEER – March 16, 2021

Memphis, Tenn. (March 16, 2021) – The National Institute of Diabetes and Digestive and Kidney Diseases recently awarded Jonathan Wall, PhD, professor in the University of Tennessee Health Science Center’s Graduate School of Medicine in Knoxville, a $1.79 million grant for his study titled “Developing a Theranostic Immunotherapy for Systemic Amyloidosis.”

Read the full article here.


DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) Becomes the First FDA-Approved Treatment for Patients with Newly Diagnosed Light Chain (AL) Amyloidosis

DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) Becomes the First FDA-Approved Treatment for Patients with Newly Diagnosed Light Chain (AL) Amyloidosis

January 15, 2021 (HORSHAM, Pa.) – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the U.S. Food and Drug Administration (FDA) approval of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.1 DARZALEX FASPRO® is the first and only FDA-approved treatment for patients with AL amyloidosis.

Read the complete press release here.


Intellia Therapeutics Receives Authorization to Initiate Phase 1 Clinical Trial of NTLA-2001 for Transthyretin Amyloidosis (ATTR)

Intellia Therapeutics Receives Authorization to Initiate Phase 1 Clinical Trial of NTLA-2001 for Transthyretin Amyloidosis (ATTR)

Oct 19, 2020

NTLA-2001: First single-course therapy that potentially halts and reverses ATTR

On track to dose first patient by year-end with a systemically delivered CRISPR/Cas9-based therapy

CAMBRIDGE, Mass., Oct. 19, 2020 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate its Phase 1 study, which will evaluate NTLA-2001 for the treatment of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).

Read more here


Subcutaneous Daratumumab Plus CyBorD Improves Outcomes in AL Amyloidosis

Subcutaneous Daratumumab Plus CyBorD Improves Outcomes in AL Amyloidosis

Raymond Comenzo, MD, Tufts Medical Center, Boston, Massachusetts, discusses results from the ANDROMEDA trial, which showed superiority with the addition of daratumumab to CyBorD over CyBorD alone for patients with light chain (AL) amyloidosis. These results were presented during the virtual 2020 EHA Congress.

View the video here




Genetic Disease More Common Among African Americans Can Now Be Treated

Genetic Disease More Common Among African Americans Can Now Be Treated

Brandpoint (BPT) – Posted: Wednesday, December 11, 2019 4:31 am

(BPT) – A disease called hereditary ATTR amyloidosis (hATTR) is linked to a genetic mutation that has been passed through generations of Americans with ancestors from West African countries such as Ghana, Sierra Leone, Cote d’Ivoire, and Nigeria. hATTR amyloidosis is difficult to diagnose and often missed by doctors and patients because its symptoms and related complications, which include numbness, tingling or loss of sensation in the hands and feet, dizziness, and fainting, are similar to those of common conditions such as diabetes and hypertension (high blood pressure). But unlike these conditions, which are frequently recognized and can be managed through a combination of lifestyle factors and a variety of available medicines, hATTR amyloidosis is a complex, multi­-system disease that worsens over time if not addressed. Today, accurate genetic tests that diagnose hATTR amyloidosis are readily available, so early action is important.

 Click here to read the full article.


National study seeks earlier diagnosis of ATTR cardiac amyloidosis in minorities

National study seeks earlier diagnosis of ATTR cardiac amyloidosis in minorities

NEWS RELEASE 3-SEP-2019

Boston Medical Center and Columbia University Irving Medical Center awarded grant for new trial

BOSTON – Researchers at Boston Medical Center (BMC) and Boston University School of Medicine (BUSM), in collaboration with Columbia University Irving Medical Center (CUIMC), are leading a national, multi-site study aimed to achieve earlier diagnosis of transthyretin cardiac amyloidosis (ATTR-CM). The National Institutes of Health has awarded a five-year, $7.2M grant to fund the Screening for Cardiac Amyloidosis with Nuclear Imaging in Minority Populations (SCAN-MP) trial.

Led by co-principal investigators Frederick L. Ruberg, MD at BMC/BUSM’s Amyloidosis Center of Excellence and Mathew S. Maurer, MD from CUIMC’s Cardiac Amyloidosis Program, the grant will enable the establishment of an important, community-based cohort of older minority patients with congestive heart failure. A total of 800 participants will be recruited from BMC/BUSM in Boston and the CUIMC, NYP-Allen and Harlem Hospitals in New York City.

Read full article here.


U.S. FDA APPROVES VYNDAQEL® AND VYNDAMAX™ FOR USE IN PATIENTS WITH TRANSTHYRETIN AMYLOID CARDIOMYOPATHY, A RARE AND FATAL DISEASE

U.S. FDA APPROVES VYNDAQEL® AND VYNDAMAX™ FOR USE IN PATIENTS WITH TRANSTHYRETIN AMYLOID CARDIOMYOPATHY, A RARE AND FATAL DISEASE

— FIRST AND ONLY MEDICINES APPROVED FOR PATIENTS WITH EITHER WILD-TYPE OR HEREDITARY TRANSTHYRETIN AMYLOID CARDIOMYOPATHY —

Monday, May 6, 2019 – 6:45am EDT

Pfizer Inc. (NYSE:PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved both VYNDAQEL® (tafamidis meglumine) and VYNDAMAX (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are two oral formulations of the first-in-class transthyretin stabilizer tafamidis, and the first and only medicines approved by the FDA to treat ATTR-CM.

For more information, click here