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…
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…
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.…
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…
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)…
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…
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…
CRISPR (clustered regularly interspaced short palindromic repeats) was discovered over a decade ago by Emmanuelle Charpentier and Jennifer Doudna, two scientists who recently shared the Nobel Prize. Scientists using…
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…
Currently, the European Hematology Association (EHA) Virtual Congress 2021 is going on from June 9 through 17, 2021. During the Congress, hematologists and other stakeholders will discuss clinical research and…
For more than 30 years, Ionis Pharmaceuticals, Inc. ("Ionis") has worked to create RNA-targeted therapies for patients in need. Now, according to a recent press release, the company initiated…
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