Two of Intellia Therapeutics’ clinical trials that had been on hold since October 2025 have now been released by the FDA. The Agency initially issued the holds due to reports…
As reported on Pharmaceutical Business Review, Affinia Therapeutics has been granted orphan drug designation by the US Food and Drug Administration (FDA) for AFTX-201, an experimental gene therapy being developed…
As reported on BioSpace, BridgeBio Pharma has reported new findings suggesting that its transthyretin (TTR) stabilizer, Attruby (acoramidis), may help preserve kidney function in patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM),…
As reported on Cardiovascular Business, new long-term findings presented at the American College of Cardiology (ACC) 2026 meeting provide compelling evidence that early and continuous treatment with acoramidis (Attruby) confers…
My journey with cardiac amyloidosis started shortly after I retired as a firefighter/paramedic in spring 2018. I started with trigger fingers—one in 2018 and two more in 2020. Also in…
My name is Faye. I’m blessed to be the wife of the love of my life, Brad, and the proud mom of two extraordinary boys we adopted in 2013—now teenagers…
Two of Intellia Therapeutics’ clinical trials that had been on hold since October 2025 have now been released by the FDA. According to The New England Journal of Medicine, the…
Light chain amyloidosis (AL) is a progressive and rare disease whereby abnormal plasma cells found in the blood and urine over-produce light chain proteins. These proteins misfold into amyloid fibrils…
Nexiguran ziclumeran (nex-z), an investigational CRISPR-Cas9 gene therapy for transthyretin amyloid cardiomyopathy (ATTR-CM), continues to demonstrate long-term efficacy in early-phase research, even as its phase III trials remain on hold…
Written by Diane Wilkie My hATTR story began in the mid-1960’s when my father, a healthy, strong, outdoorsy guy in his late 40’s started tripping, experiencing numbness, and having dizzy…
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an infiltrative heart disease driven by protein instability. Normally a tetramer, transthyretin can dissociate into monomers that misfold, aggregate, and deposit as amyloid fibrils within…
A new potential treatment for light chain amyloidosis is making significant strides in clinical development. According to a recent update reported by Drugs.com, the phase III clinical program called “CARES”…
Fredericksburg, Virginia resident Johnny Boatman is a former national guardsman who had been in excellent physical condition all his life. That was until 2022 when he felt a sharp pain…
Up to 10% of heart failure cases could be caused by cardiac amyloidosis. Yet amyloidosis remains underdiagnosed and misunderstood. Broadening our understanding of this rare disease could contribute to earlier…
A new global study (THAOS NCT00628745) of over 6,000 people who had been diagnosed with ATTR amyloidosis found that almost one in every four patients exhibited cardiac and neurological symptoms.…
2nd Annual Bradley Z. Naifeh Amyloidosis Conference March 2, 2024 Houston, TX Presented by the Bradley Z. Naifeh Amyloidosis Clinical Research and Treatment Program at Houston Methodist J.C. Walter Jr.…
2024 Light the Night for Amyloidosis Month March 1-31, 2024 "Light the Night for Amyloidosis" is an awareness campaign created to draw attention to amyloidosis symptoms, diagnosis, treatment, and hopefully a…
In 2018, the U.S. Food and Drug Administration (FDA) approved Onpattro (patisiran) as a treatment for polyneuropathy in people with hereditary transthyretin-mediated (ATTR) amyloidosis. Onpattro is an intravenously administered,…
Prothena Corporation which is based in Dublin, Ireland recently announced, via Business Wire and Biospace results of the VITAL Phase 3 clinical trial showing a significant survival benefit…
On March 9, 2023, Issues Concerning Athletes held its 13th Annual Celebrity Bartender Night at Wasted Grain in Scottsdale, AZ. Erica Brooks, the Founder of Issues Concerning Athletes, spearheaded the…
According to a story on pharmaphorum.com, the UK's National Institute for Health and Care Excellence (NICE) has given a positive opinion for the drug vutrisiran (marketed as Amvuttra) as a…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In June 2022, the FDA approved Amvuttra, a prescription medicine designed to treat polyneuropathy (nerve damage) due to hereditary transthyretin amyloidosis (hATTR amyloidosis). This approval was huge. Typically, managing…
It can sometimes be difficult to spur research and drug development within the rare disease space. For this reason, the Orphan Drug Act was created. The Orphan Drug Act provided…
In a news release from biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. ("Alnylam"), the company shared that positive data was available from the Phase 3 APOLLO-B clinical trial.…
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