According to a post from gaucherdisease.org, clinical trials are an essential component to the development and testing of new therapies for Gaucher disease and other rare diseases. For some rare…
.jpg)
Alzheimer’s disease (AD) is a neurodegenerative disease and the major cause of impaired memory affecting the older population. Reported cases are rapidly increasing. So far, clinical trials have failed…
.png)
Fierce Biotech recently focused on a report by The BMJ, a British science journal, on the validity of clinical trials which are critical to decision making by patients and…
Tay-Sachs Disease September is Tay-Sachs Disease Awareness Month. Voted on by the Senate unanimously in 2008, this month aims to spread awareness about this disease and the importance of carrier…
According to a recent publication from the Duke University School of Medicine, Dr. Rodger Liddle believes Parkinson's disease may have origins in an unlikely place — the gut. Liddle, a…
According to a story from scienmag.com, a pair of five year grants that were awarded to Dr. Mark Zylka will go towards projects focused on autism and the rare disease…
According to a story from clinicaltrials.gov, the Children's Hospital of Philadelphia is sponsoring a study that has the potential to make mitochondrial disease research and drug development much easier. The…
Genes themselves are a relatively new discovery, found only about a century ago. Gene therapy, a medical procedure that alters genes to address disease, is a concept a few decades…
A recent publication from BMC Pediatrics outlines a case study of Kawasaki disease in a group of infants from Italy. Because Kawasaki disease is a rare disease (with only an…
According to a story from ANCA Vasculitis News, a recent study from Spain is highlighting the need for an updated classification system for ANCA-associated vasculitis that can predict patient prognosis…
A recent study, published in Nature, examined whether or not stem cells could be modified to reduce their chance of rejection. Stem cell therapies are utilized in a wide array…
According to a publication from BioSpace, researchers from Michigan's Memorial Healthcare Institute for Neurosciences and Multiple Sclerosis are taking full advantage of recent technological advances in neurofilament light chain testing…
Bob Hussey Bob Hussey is a 62-year-old mechanical engineer who, thanks to his own experience as an ulcerative colitis patient, has created a new invention that could help a wide…
According to a story from Newswise, a recent phase 2 clinical trial testing a combination treatment for the rare cancer rhabdomyosarcoma has displayed encouraging results. The combination consists of the…
According to a story from Science Codex, a study conducted by a team of researchers associated with the Nemours Children's Health System is emphasizing the benefits of genetic analysis for…
According to a story from sectorpublishingintelligence.co.uk, studies utilizing data gathered in the real-world treatment setting are highlighting the characteristics of two different therapies for multiple sclerosis (MS) developed by the…
According to a story from Healio, the results of a randomized trial suggest that patients with eosinophilic esophagitis are likely to experience rapid relapse if they stop treatment. This was…
According to a story from Science Daily, the University of California San Francisco's Benioff Children's Hospitals have successfully treated an infant with juvenile myelomonocytic leukemia. The drug used for treatment…
According to a story from Medscape, a recent study is highlighting the fact that many thyroid cancer survivors are still worried about cancer-related problems years after the conclusion of treatment.…
According to a story from wearegreenbay.com, a team of scientists associated with the University of Wisconsin, Madison are undertaking a project that will help improve the effectiveness of research related…
According to a story from Angioedema News, a recent study has found that the drug icatibant (marketed as Firazyr) can safely treat older patients with hereditary angioedema, a rare condition…
According to a story from newswise.com, the American Neuromuscular Foundation has recently awarded a grant to Dr. Karissa Gable. This grant will go towards a research project headed by Dr.…
According to a story from Angioedema News, the results of a recent study have led authors to conclude that genetic testing can be useful for the diagnosis of hereditary angioedema…
According to a story from journals.lww.com, The Lazarex Cancer Foundation and the University of California San Francisco have announced plans to work together on a three year study that will…
According to a story from Biotech 365, the biotechnology company Viela Bio announced the publication of critical results from a recent study that tested the company's investigational drug inebilizumab as…
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.
