About BridgeBio Pharma BridgeBio Pharmaceuticals was created in 2015 by veterans from the fields of academia and biotechnology. The company focuses on the development of new therapeutic treatments for genetic…
Multiple Myeloma Multiple Myeloma is a rare form of cancer that develops in the body's plasma cells. These cells are normally responsible for helping the body fight infection. The accumulation…
About Graft-Versus-Host-Disease (GvHD) Graft-Versus-Host-Disease (GvHD) occurs when a patient's body rejects cells which have been transplanted. For instance, it may develop following a stem cell transplant or a bone marrow transplant. The…
The Problem One of the greatest challenges in the investigation of nanoscale treatments for people with rare diseases is figuring out how to administer the therapy to the correct part…
According to a press release from X-Rx Inc., a privately-held American biotechnology company, the Food and Drug Administration has accepted the company's Investigational New Drug (IND) application for X-165, an…
deCODE Genetics, which is a research subsidiary of Amgen genomics based in Iceland, has recently created an extremely detailed genetic map. They wanted to uncover more about the human genetic…
What is Duchenne Muscular Dystrophy? Duchenne muscular dystrophy (DMD) is a rare genetic condition that is ultimately fatal. It causes muscles to weaken and usually results in heart failure before age…
There is a high unmet need to find more effective treatments for liver cancer. Currently, treatment options for patients often only increase life expectancy by 3 to 4 months. An accidental…
Hope everyone's week is going well! Today, we're highlighting an article discussing the difficulties of securing a CIDP diagnosis, followed by an update on a Niemann-Pick Type C treatment. Next,…
According to a story from PR Newswire, The Lustgarten Foundation, which is the largest private organization that funds research on pancreatic cancer, has recently announced that opening of its fourth…
According to a story from finanznachrichten.de, the Swedish drug company Calliditas Therapeutics has recently announced that the US Food and Drug Administration (FDA) has given one of the company's experimental drugs…
According to a story from Rare Disease Report, the US Food and Drug Administration (FDA) has announced its approval of a combination therapy from Janssen which consists of ibrutinib plus…
Mendelian Mendelian is a healthtech company in London whose primary goal is to help the National Health System (NHS) diagnose rare diseases faster. With 3 million people currently living with a…
Rett Syndrome Rett syndrome is a rare neurodevelopmental disorder. It affects females disproportionately, at a far greater rate than males. It causes cognitive, autonomic, and neurological dysfunction as well as a loss of…
This is the story of Tom Dinwiddie, who was diagnosed with terminal cancer on April 2, 2012. This year, now age 69, he is officially six years cancer free. The Diagnosis…
According to a story from BioSpace, the biotechnology company REGENXBIO, Inc. recently announced that its experimental gene therapy candidate called RGX-181 has been granted Rare Pediatric Disease designation from the…
What is osteogenesis imperfecta? Osteogenesis imperfecta (OI) actually refers to a group of four distinct rare disorders- OI Type I, II, III, and IV. They're all characterized by brittle bones which…
Thanks to a chance encounter in a bike shed, two research groups at the Francis Crick Institute have discovered two distinct ways that pancreatic cancer tumors form and grow. The first research…
A recent study, called TOPCAT, evaluated the effects of spironolactone as a treatment for individuals living with Chronic Kidney Disease (CKD) who are also diagnosed with heart failure with preserved ejection fraction,…
Familial Chylomicronemia Syndrome (FCS) Is a rare disease caused by malfunctioning lipoprotein lipase. This results in a buildup of triglycerides in the body's plasma. FCS can cause pancreatitis, memory loss, nerve…
Time for Revision Like many things in this world, research for rare diseases such as Spinal Muscular Atrophy (SMA), Multiple Sclerosis, and Duchenne Muscular Dystrophy (DMD) has evolved substantially in recent years. This evolution…
AL Amlyoidosis is a rare disease which causes a buildup of harmful proteins (amyloids) in organs such as the kidneys and the heart. This accumulation causes organ damage as well as…
According to a story from arkansasonline.com, Rep. Julie Mayberry R-Hensley of the Arkansas General Assembly is sponsoring House Bill 1074. This legislation proposes adding the rare and potentially fatal genetic…
Hereditary angioedema (HAE) is a rare condition which causes severe swelling in various areas of the body. Takhzyro has been approved as a treatment for this disease. The drug was created…
According to a story from the Tenterfield Star, a fundraiser was recently held for Tommy Johnston, a one year old boy who was diagnosed with the rare Niemann-Pick disease type…
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