According to a story from businesswire.com, the Bristol Myers Squibb Company recently released the topline results from its phase 3 clinical trial. This study was testing a combination treatment consisting…
In a study published recently in the Orphanet Journal of Rare Diseases, a team of researchers identified distinct variations in the neuroanatomical and neuropsychiatric phenotypes of cystinosis patients. Phenotype refers to visible,…
According to a story from mdmag.com, a recent proof-of-concept style study suggests that a certain machine learning tool could be of benefit to patients living with the rare condition alkaptonuria.…
The term ‘failure’ may no longer apply to cancer vaccines according to a recent article in Marketwatch. Researchers have endured over four decades of frustration since the first two…
An issue with current treatments for Parkinson's disease is that they only provide temporary relief from symptoms. There are no therapies that halt or even slow the progression of the…
Rare Disease Institute The Rare Disease Institute (RDI) is a new initiative aimed at improving collaboration between rare disease stakeholders. The aim is to facilitate faster diagnosis and better care…
The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…
According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…
As originally reported in Biospace, two research networks dedicated to forming bridges between rare disease research around the world will share their niches to promote discovery for rare diseases. TriNetX…
Why are rare diseases so difficult to study? Is it just their rarity? Or is it also industry challenges? Unfortunately, industry has a lot to do with it. Clinical Trial…
Clinical trials are an opportunity for patients to try new treatments and medicines that offer the potential to improve or extend their lives. The number of clinical trials have…
Lyme disease is the most common vector-borne disease, and it has become more prominent due to climate change and urban developments spreading further into wooded areas. Treatment is typically effective…
Clinical trials and healthcare as a whole are being transformed by new technology. It's essential for clinical trial sponsors to be aware of the new options available to them, and…
Alyla Annac When Alyla Annac was just 19 years old her father passed away from heart failure. He had suffered 3 heart attacks and doctors said there was nothing else…
Christine Wright Christine Wright was an active, athletic, healthy mother of three. But in 2007 she started exhibiting strange symptoms that no one could give her an answer to. She…
According to a story from PR Newswire, scientists are making significant progress in researching new treatments for glioblastoma, a rare and deadly brain cancer. A variety of drug companies are…
During the 1990s, researchers had a dream that by 2020, every individual would have access to their own genome. This would aid in their diagnosis and treatment as soon as…
Without understanding the cause of disease it is difficult to understand how to most effectively care for patients. Instead of addressing the underlying cause of disease, physicians are forced to…
Teva Pharmaceuticals recently finished two clinical trials of their Tourette syndrome treatment, deutetrabenazine. These two studies, titled ARTISTS 1 and ARTISTS 2, did not meet their primary endpoints, as they…
By Danielle Bradshaw from In The Cloud Copy Neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (pNFs) is a condition that many children struggle to manage due to a lack of proper…
For years scientists have tried to understand how and why tau, well known to researchers for its implication in Alzheimer’s disease, changes from its normal form to a harmful…
By Jodee Redmond from In The Cloud Copy A recent study suggests that certain antibodies could be linked to specific human leukocyte antigen (HLA) genes and environmental components in scleroderma.…
By Danielle Bradshaw from In The Cloud Copy A Tufts University science team was able to identify a means to possibly reverse Friedreich’s ataxia. Friedreich’s ataxia (also known as…
By Natalie Homan from In the Cloud Copy Most people have heard of type 1 and type 2 diabetes. Many do not know, however, that there is an “intermediate” form…
According to a story from Financial Buzz, the ophthalmology company Acucela Inc. has recently announced an update on the progress of its phase 3 clinical trial. This clinical trial is…
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.
