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.…
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…
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…
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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 National Gaucher Foundation, a significant challenge for many patients with Gaucher disease are the effects that the illness can have on bones. Common treatments…
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…
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).…
According to a story from Rocket News, the class of drugs known for their "gene silencing" mechanism of action appear to be effective in treating porphyria, a rare disease that…
Biophosphonates (BPs) are common treatments for osteoporosis and metastatic bone cancer. They help to prevent bone loss. Unfortunately, these drugs can have serious side effects. One of the most severe…
Danon Disease Danon disease (DD) is an extremely rare condition that is not very well understood in the medical community. It was first described in 1981, and it is named…
A three-year research grant of $11 million has been awarded through the Gilbert Family Foundation in America. According to a recent article in News-Medical Life Sciences, the Foundation was formed…
According to a story from finanzen.at, the biotechnology company Orbsen Therapeutics recently announced that its immunotherapy product ORBCEL-C is to be tested in a multi-site clinical trial called MERLIN that…
People living with rare diseases such as amyotrophic lateral sclerosis (ALS) or muscular dystrophy as well as those who have suffered from a stroke or spinal cord injury often experience…
According to a story from Xconomy, a recent report from the Institute for Clinical and Economic Review (ICER), based on Boston, is calling for reductions to the price tags of…
According to a recent article in Check Orphan, genetic and biochemical testing are combined in a new method of metabolic testing called the CentoMetabolic panel. The announcement comes from…
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