According to a story from globenewswire.com, the gene therapy company Fibrocell Science, Inc. and the biopharmaceutical company Castle Creek Pharmaceuticals have recently announced a collaboration between the two companies. The…
According to a report from ArsTechnica, scientists believe that some people might have medicinal poop. What might sound like a bad punchline to a child’s joke is actually the result…
According to a story from Top Class Actions, the drug companies Biogen, Inc. and Abbvie recently released a recall for their multiple sclerosis drug Zinbryta following several reported cases of…
Results from the largest United States study to date of Lambert-Eaton myasthenic syndrome (LEMS) have indicated that for the majority of patients, Firdapse is the most effective treatment option currently…
Researchers at GENFIT are excited about promising results from studies investigating a new potential therapy for PBC and NASH, two rare liver diseases. Primary Biliary Cholangitis Primary biliary cholangitis (PBC)…
According to a story from EurekAlert!, a group of researchers affiliated with Penn Medicine and Children's Hospital of Philadelphia recently conducted an experiment in which the scientists used CRISPR gene…
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Although guidelines for Parkinson’s patients suggest that they visit a specialist every six months, according to a report in Parkinson’s News Today, the waiting period may be up to…
According to a story from inpublic.globenewswire.com, the Novartis company AveXis has released interim data from an ongoing Phase 3 clinical trial of Zolgensma, a gene therapy drug that is being…
Lyme disease is an infection by a type of bacteria called a spirochete. This type of bacteria is carried by deer ticks and when an infected tick bites an…
According to a story from globenewswire.com, the biopharmaceutical company TG Therapeutics, Inc. recently made an announcement declaring that the US Food and Drug Administration (FDA) had awarded the company Orphan…
When you’re diagnosed with a rare condition it can change the entire course of your life, at least at the beginning. But those living with diseases like these are fighters.…
According to a publication from KYN24, the Food and Drug Administration recently approved mucopolysaccharidosis type VII (MPS VII) drug Mepsevii for marketing and administration to patients in the United States.…
Severe Combined Immunodeficiency Severe Combined Immunodeficiency (SCID) is a rare disease which causes patients to practically have no immune system at all. That means that for these patients, even a…
According to a publication at BioPortfolio, Belgian researchers are developing an orally-administered antibody medication for patients living with gastrointestinal disorders. In most cases, antibodies are injected into the bloodstream for…
A recent publication from Charcot-Marie-Tooth News announced that this year, for the first time ever, the Muscular Dystrophy Association will be hosting its annual clinical conferences at the same event…
Lithium boosts muscle strength in mice with rare muscular dystrophy New drug target identified Click to share on Pinterest (Opens in new window) Removing one gene caused normal muscle muscle…
Alex Knoll Alex Knoll is not your average 14-year-old. In addition to being a student, he's also an inventor, entrepreneur, app creator, and public speaker. Most of the Idaho resident's…
Madison Ricci Madison Ricci is a 19-year-old who was diagnosed with Lupus at the age of 12. Lupus is a rare disease in which the body’s immune system begins attacking…
According to a story from Charcot-Marie-Tooth News, early preliminary testing of a possible gene therapy treatment for a certain type of Charcot-Marie-Tooth disease showed potential in a mouse model. This…
A press release from American biopharmaceutical company AMO Pharma, published by PR Newswire, detailed the recent completion of the Company's new congenital myotonic dystrophy type 1 (CDM1) symptom-evaluation scale. The…
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The mesothelioma diagnosis was a surprise to Raya Bodnarchuk of Baltimore, Maryland because she had never worked in a factory, mine, the military, or a shipyard. These are the typical…
According to a story from the Daily Record, cystic fibrosis patient Kelli Gallacher found that she was able to stabilize her disease using the drug Symkevi. Now that her own…
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A birth involving mitochondrial replacement therapy took place as part of a clinical trial in Greece according to a recent article in STAT. Greece is now the second country…
According to a story from the National Gaucher Foundation, a significant challenge for many patients with Gaucher disease are the effects that the illness can have on bones. Common treatments…
According to a story from news-medical.net, the process that causes the neurons responsible for voluntary muscle movement in amyotrophic lateral sclerosis (also known as Lou Gehrig's disease) to die is…
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