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…
LLUCH Loma Linda University Children's Hospital (LLUCH) is one of six hospitals in the University's health system. They are a faith-based system located in Southern California. They are the only…
According to a story from globenewswire.com, Vertex Pharmaceuticals Inc. and CRISPR Therapeutics have recently announced that the US Food and Drug Administration (FDA) has awarded CTX001, an experimental gene-edited stem…
Alnylam Pharmaceuticals has been developing lumasiran as a potential treatment for primary hyperoxaluria type 1 (PH1). This investigation began with ILLUMINATE-A, a clinical trial whose results should be announced by…
According to a story from EurekAlert!, a new mathematical approach pioneered by a team of scientists from New York University and Cincinnati Children's could greatly improve the analysis of scientific…
Personalized medicine is the latest craze in patient care. In essence, it refers to the development of treatment options which take into account the patient as an individual. It reduces…
According to a story from BBC, Louise Moorhouse gets the nutrition that she needs through two sources: pills and foul tasting nutrient shakes. This is because she was born with…
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Familial Hypercholesterolemia (FH) is often misdiagnosed as plain old high cholesterol because of overlapping symptoms such as elevated lipid levels. According to a recent article in MedicalView, FH is three…
According to a story from Pulmonary Hypertension News, the drug developer United Therapeutics recently issued a statement in which the company announced that it was ceasing the development of esuberaprost…
Happy Thursday! We hope everyone's month is going well! This week, we're highlighting two stories about drug pricing and reimbursement. Next, we have an article on mastocytosis diagnoses and (yet…
Upcoming Conference The International Society for Mannosidosis & Related Diseases (ISMRD) serves as an international patient advocate for those living with glycoprotein storage diseases. These include alpha-mannosidosis, fucosidosis, mucolipidosis type…
According to a story from AJC, three year old Reid Peteet is one of over 550 kids that have been diagnosed with acute flaccid myelitis since 2014. 2018 saw a…
For years FibroGen has been investigating a drug called pamrevlumab. They believe it could be an effective treatment for Duchenne muscular dystrophy (DMD), pancreatic cancer, and idiopathic pulmonary fibrosis (IPF).…
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