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…
.png)
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).…
According to a press release from American biotechnology company Intercept Pharmaceuticals, the company recently released additional data supporting its phase 3 clinical trial of obeticholic acid (OCA) in the treatment…
Six weeks ago, Gretchen and Josh of Oklahoma rushed their precious 5-month-old Opal to the hospital after she became extremely sick from a cold. It didn't take doctors too long…
April 15th is International Pompe Disease Day! The International Pompe Association (IPA) is an organization of Pompe disease patient groups that seeks to coordinate activities and share experiences worldwide. The…
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…
It's one thing to cope with a new rare disease diagnosis. It's another entirely to cope with that diagnosis and then four years later learn that diagnosis is incorrect and…
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 CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy and Duchenne muscular dystrophy. These…
According to a press release from Inovio Pharmaceuticals, the American biotech company's experimental recurrent respiratory papillomatosis (RRP) drug INO-3106 showed promising results at the end of a pilot clinical study…
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…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
