Philippe Pakter’s daughter Lysiane was born on March 29th 2017 in Lyon, France. However, soon after her birth, Philippe and his partner Delphine were hit with shocking news: Lysiane was…
Happy Thursday, Patient Worthy Friends! This week we're highlighting two stories on families who went on a diagnostic odyssey to understand their children's rare diseases. Additionally, we have some big…
According to a story from The People's Pharmacy, the US Food and Drug Administration (FDA) has given the green light for the use of generic drugs manufactured in India despite…
According to a story from ucb.com, the biopharmaceutical company UCB has recently released the final results from its phase 2 clinical trial which tested the company's investigational monoclonal antibody rozanolixizumab…
By Jack Gerard from In The Cloud Copy Travis Allen, of Lisbon, Iowa, was a painter who specialized in photorealistic works. Unfortunately, a rare autoimmune condition stole his eyesight…
According to a story from the Washington Post, rare diseases are a much more widely spread problem than you might think. There are at least 7,000 diseases that are considered…
By Rachel Whetstone from In The Cloud Copy. Rheumatoid Arthritis (RA) causes pain and suffering to patients around the globe, and it can be difficult to diagnose in the early stages.…
The European Organization for Research and Treatment of Cancer (EORTC) has been evaluating zotiraciclib, which is a new treatment for glioma. This drug has recently been granted the Orphan…
Jojo, a ten-year-old girl, has become the first person to ever receive a new gene therapy for the treatment of GM1 gangliosidosis. The treatment, called AXO-AAV-GM1, was used in…
As originally reported in Angelman Syndrome News, Terry Jo Bichell is a PhD researcher, former director and scientific officer of the Angelman Biomarkers and Outcomes Measures Alliance and Tennessee's ambassador…
By Jack Gerard from In The Cloud Copy. While vaccines are largely considered safe for most of the population, there are some people whose doctors recommend avoiding at least some…
By Jodee Redmond from In The Cloud Copy The results of a study have found that betaine and the active form of folic acid, L-methylfolate, are not effective measures to…
Pulmonary Hypertension Association The Pulmonary Hypertension Association (PHA) is headquartered in Silver Spring, Maryland. It was formed in 1991 by 4 women passionate about finding a cure. Additionally, they hope…
Copyscape score: 6% Currently there are only two FDA approved treatments for the treatment of amyotrophic lateral sclerosis (ALS). These treatments are only modestly effective, partly because ALS is not…
As originally reported in Medical Xpress, CRISPR has been a hot conversation starter in recent years. It has been framed as gene editing made easy: cheaper and simpler than previous…
According to a story from BioPortfolio, Acceleron Pharma and the Bristol-Myers Squibb Company have announced recently that the US Food and Drug Administration (FDA) will review the supplemental Biologics License…
The iPain Summit 2019 took place from November 14th-16th, 2019. Patient Worthy had the privilege to attend this event and cover it live on social media. This summit was focused…
At the International Pain Foundation's iPain Summit, held this year from November 14th-16th, a wide variety of topics related to the challenges faced by patients with chronic pain were discussed.…
As reported in the Guardian, at Bath University, scientists have created a groundbreaking technology for the future of medication: artificial nerve cells or neurons created on silicon to replace deteriorating…
Happy Thursday, Readers! This week we're highlighting four articles on rare disease and chronic illness patients and ways to provide support that goes beyond treating physical symptoms. First, we have…
According to a story from BioPortfolio, the biopharmaceutical company Incyte Corporation has recently announced that the company's New Drug Application (NDA) was recently accepted by the US Food and Drug…
Contact: Britta Vander Linden bvanderlinden@everylifefoundation.org 917.604.6518 30 million Americans need more treatment and diagnostic opportunities https://everylifefoundation.org/rare-disease-community-calls-on-congress-fda-to-enact-life-saving-public-policy-solutions/ (Washington, D.C., December 4, 2019) Hundreds of rare disease advocates from around the country were brought to Washington,…
Getting a diagnosis for a rare disease is hard. Not many people are familiar with the many obscure symptoms and manifestations of these diseases. Because of this, patients with rare…
As originally reported in Medscape Neurology News, for the last few years, doctors and researchers have been excited by the new prospect to use an obesity drug, fenfluramine, to treat…
As originally reported in Mirror, when Sian Hopper was in secondary school, she began to get headaches. She attributed them to stress: she had been studying for her GCSE exams,…
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