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…
The latest statistics on heart disease and its effect on Black communities are startling. According to an article in the Dallas Examiner, Black people are fifty to seventy percent more…
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.…
From June 11th until the 14th, the Society of Nuclear Medicine & Molecular Imaging (SNMMI) Annual Meeting was held in Vancouver, Canada. At this meeting, Attralus Inc. shared new data…
It is no secret that there is an inequity when it comes to race and medical research. More often than not, research does not take all races and ethnicities into…
On March 29, 2022, the International Waldenström's Macroglobulinemia Foundation (IWMF) hosted a webinar titled "An Overview of Amyloidosis and WM with Dr. Morie Gertz." The goal of this program was…
In August of 2021, Intellia Therapeutics of Cambridge and its partner Regeneron Pharmaceuticals published a study reported in the Boston Globe, showing results of a single treatment using CRISPR-Cas9 technology.…
Diagnosing certain conditions can be difficult for many reasons, one of which is the similarity between symptoms of numerous diseases. Doctors can mistake one disease for another, which then impacts…
The FDA has just announced that they have granted Orphan Drug Designation to an investigative treatment called eplontersen. This therapy is being studied as a treatment option for transthyretin (ATTR)…
A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…
AstraZeneca has entered into two collaborative agreements which will improve research for two rare diseases: transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloidosis (ATTR). N1006 The first collaboration is with Neurimmune…
Diagnosing amyloidosis (abnormal protein in tissue) is a complex process. Over eighty percent of amyloidosis patients are undiagnosed. The disease takes hold after the blood plasma cells in the bone…
A recent article in the Northern Kentucky Tribune highlights a rare condition that has been exposed by new technology. Cardiologists have found that many patients diagnosed with heart failure are…
CRISPR Cas9 gene editing is still looked upon as a new phenomenon in medicine. Yet it appears in the news almost every week. This week Neurology Today shines a light…
According to a recent article, investigators created a machine learning model in order to identify if patients are at risk of rare cardiomyopathy. Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin amyloid cardiomyopathy…
When it comes to advances in hereditary ATTR amyloidosis (hATTR amyloidosis), biopharmaceutical company Alnylam Pharmaceuticals, Inc. ("Alnylam") has been on top of its game. Alnylam helped develop Onpattro and is…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
On August 9, 2021, biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (“Alnylam”) shared that enrollment is now complete for the Phase 3 HELIOS-B clinical trial. During the trial, researchers…
Currently, there are no treatments available for patients with hereditary ATTR amyloidosis (hATTR); most available therapies are designed to aid with symptom management. However, biotechnology company and RNA-targeted therapeutics leader…
Amyloidosis is a rare disease which can cause organ failure. It is the result of an abnormal buildup of amyloid proteins in the blood. These proteins are then deposited within…
CRISPR is a gene-editing technology which holds the potential to treat a variety of genetic diseases and improve patient outcomes. Overall, CRISPR works by creating precise cuts and edits within…
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