In very recent news, the biopharmaceutical company Chiasma, Inc. announced that it has come to an agreement with the US FDA regarding the design of a phase III clinical trial…
If you or someone you love has been diagnosed with idiopathic pulmonary fibrosis (IPF), it's easy to become discouraged by the lack of medical options. Although almost 50,000 people a…
In a study at the University of North Carolina (Chapel Hill), researchers found that there may be a much more effective new way to isolate lung stem cells in order…
Hereditary Angioedema, or HAE, is an extremely rare genetic condition. Patients with HAE experience swelling under the skin in possibly many different parts of the body. Individuals with HAE do…
In 1901, a German ophthalmologist named Karl Stargardt reported the first case of macular degeneration in a child. Today, we know this is very rare in children. Approximately one in…
Patient Worthy’s very own contributor Iva Rauh, with a tiny nudge from Patient Worthy, attended the International Pemphigus Pemphigoid Foundation’s Conference in Lubeck, Germany. Who better to learn about the latest developments than…
Recent findings from research done at the University of Pennslyvania indicate a new biomarker associated with a group of rare metabolic diseases called mucopolysaccharidoses (MPS). A biomarker is a measurable…
A new study shows that two specific compounds found in common sunscreen are actually effective as multiple sclerosis symptom suppressors. These two substances are salate derivatives and are also part…
August 21st is the online registration deadline for this rare disease event! The site says the 15th and the 21st, so either way, you'll want to check out the details…
A North Carolina-based research group has just developed research that indicates a potential stem cell treatment for lung fibrosis conditions such as cystic fibrosis (CF) and idiopathic pulmonary fibrosis (IPF).…
When I was in my 20s and 30s, I performed in a lot of musical theater performances— “Music Man,” “Big River,” “Carousel,” “You’re a Good Man, Charlie Brown.” I was…
According to the AKU Society, Flavia was only a few weeks old when her parents noticed that her urine had stained her diaper an unnatural brown color. Flavia and her…
Inspiration is everywhere—from the words of your favorite writer to the blooms and leaves on your morning walk. You just need to open your eyes, and breathe it in. Sometimes, inspiration just…
Sancilio recently completed enrolling children with sickle cell anemia/disease in a Phase 2 clinical trial to evaluate a new therapy called Altemia (docosahexaenoic acid). The SCOT trial, as it will…
Global Genes is hosting its annual Tribute to Champions of Hope event for the rare disease community. Here's all you need to know about it: What: A blue-tie cocktail hour and dinner event…
Hema Biologics have recently released something even cooler and more inventive than just their traditional hemophilia treatments. They are way on top of their game and know exactly how to…
Your hear that? It's the sound of the future knocking on the door! The MIT Technology Review reports about the first-known experiment to edit human embryos in the United States using a…
What should you know about Tourette syndrome? How can you explain it to your child? These are probably just a few questions you may have if your child has been…
Happy Friday! This week, we have a story that might break your heart about a woman who battled cystic fibrosis. But we also have a story that might inspire you about a…
Trigeminal neuralgia (TN), or extreme, sudden facial pain associated with the 5th cranial nerve, occurs frequently in patients who have received a multiple sclerosis (MS) diagnosis. In a survey with 8,590 respondents,…
Much like the United States’ National Organization for Rare Disorders (NORD), E-Rare in the European Union was launched over a decade ago to bring together organizations that fund research for…
According to Dr. Steven Carson, of all the autoimmune disorders that are both chronic and joint-related, Sjogren's syndrome remains the hardest to manage. Standards of care designed to guide doctors…
The United States FDA just granted Orphan Drug Designation to Napo Pharmaceuticals for Mytesi (crofelemer) in the treatment of short bowel syndrome (SBS). SBS is a rare disorder in which…
Orphan drugs, for those of you who don’t know, are those designated especially for rare diseases. They respond to a public health need, but not necessarily an economic need by…
In recent news in the oncology community, the FDA has approved orphan drug designation to a new drug, SurVaxM, for glioblastoma. Glioblastoma is a rare type of brain cancer that…
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