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.


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




Amyloidosis Symposium at Washington University in St. Louis

Amyloidosis Symposium at Washington University in St. Louis

The Amyloidosis Foundation is proud to be a partner at this event, Amyloidosis: A Disease Now at the Forefront of Practice Latest Diagnostic & Treatment Strategies on February 22, 2020 at Washington University in St. Louis, MO, chaired by our Board of Director, Daniel Lenihan, MD.  This is a symposium for providers, patients and their families. Continuing Medical Education (CME) credits will be available.

Patients touched by amyloidosis and their families are invited to attend the lunch and afternoon session. The breakout sessions will allow attendees the opportunity to ask questions of various medical providers and network.

Guest faculty includes:

Raymond L. Comenzo, MD
Professor of Medicine
Tufts University School of Medicine
Director, John C Davis Myeloma and Amyloid Program
Tufts Medical Center, Boston, Massachusetts

Mathew S. Maurer, MD
Arnold and Arlene Goldstein Professor of Cardiology
Professor of Medicine
Columbia University Irving Medical Center
Director, Cardiac Amyloidosis Program, New York, New York

This course is planned and designed inter-professionally by and for physicians and nurses. This live activity is designed for cardiologist, neurologist, gastroenterologist, oncologist, hematologist, nephrologist, hospitalists, internists, general medicine physicians, physician assistants, nurse practitioners, and nurses, as well as patients and their family.

The intended result of this activity is increased knowledge, competence, performance and skill and enhanced patient care. At the completion of the activity registrants should be able to:

  • State the etiology and epidemiology of both TTR and AL amyloidosis
  • Select appropriate diagnostic approach in amyloidosis
  • Differentiate AL vs TTR in diagnosis of amyloidosis
  • Outline current therapeutic options
  • Describe current research developments in AL and TTR

For more information click here

Patients and families register 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


HealthWell Foundation Opens New Amyloidosis Fund to Assist Patients with Treatment Costs

HealthWell Foundation Opens New Amyloidosis Fund to Assist Patients with Treatment Costs

GERMANTOWN, Md. – April 23, 2019 – The HealthWell Foundation, an independent non-profit that provides a financial lifeline for inadequately insured Americans, has launched a new fund to provide copayment and premium assistance to patients living with amyloidosis.  Through the fund, HealthWell will provide up to $8,000 in financial assistance for a 12-month grant to eligible patients who have annual household incomes up to 500 percent of the federal poverty level.

For more information, click here


Eidos Therapeutics Initiates ATTRibute-CM, a Phase 3 Study of AG10 in ATTR-CM with Registrational 12-month Endpoint

Eidos Therapeutics Initiates ATTRibute-CM, a Phase 3 Study of AG10 in ATTR-CM with Registrational 12-month Endpoint

February 27, 2019 07:00 ET – Source: Eidos Therapeutics, Inc.

San Francisco, Feb. 27, 2019 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced the initiation and design of its pivotal global Phase 3 trial (ATTRibute-CM) of AG10 in patients with transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). The design of the ATTRibute-CM study, which incorporates feedback from FDA, includes two potentially registrational endpoints. In Part A, benefit in change from baseline in 6-minute walk distance (6MWD) will be evaluated at 12 months, potentially accelerating the time to registration. In Part B, reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations will be evaluated at 30 months.

 

For more information, click here