By Natalie Homan from In The Cloud Copy The COVID-19 pandemic has changed the lives of millions around the world in an incredibly short span of time. It’s hard to…
By Natalie Homan from In The Cloud Copy Two businesses that advocate for patients with rare and orphan diseases announced recently that they are teaming up to help patients get…
According to Hemophilia News Today, data collected from a Phase 2b trial highlights that the subcutaneously administered DalcA is effective for the treatment of hemophilia B. Find the full press…
This was originally published as the Seventh Newsletter for the Leukodystrophy Resource Research Organisation (LRRO) on April 19th 2020. You can read more from their newsletter here. To state the blatantly…
Click here to read part 1 and here to read part 2 After coming back from the Global Genes conference, Kristie was convinced that establishing a foundation was the right…
May is recognized as Behçet's Disease Awareness Month and May 20th is recognized as Behçet's Disease Awareness Day. The focus of these events is to help spread awareness about this…
When studying biomarkers of lupus nephritis, researchers at the University of Houston discovered something interesting: race-based differences. Biomarkers are some sort of measurable substance; in this case, urinary proteins. However,…
Enanta Pharmaceuticals has recently released the results from the second phase of its INTREPID trial, which studied EDP-305 as a treatment for primary biliary cholangitis (PBC). The results were positive,…
According to CNN, the CDC recently sent out a health advisory to doctors across the United States alerting them of a new syndrome affecting children in eighteen states, Washington, D.C.,…
According to ANCA Vasculitis News, recent research shows that patients with multiple sclerosis who are being treated with Lemtrada are at greater risk of developing severe ANCA vasculitis. The drug therapy…
COVID-19 was declared a pandemic in March, marking a distinct change in daily life. With nearly 4.5 million diagnosed cases worldwide, it is no wonder people are concerned about the…
According to a story from Sanfilippo News, the healthcare tech company RDMD has recently announced that it has raised $14 million in Series A financing that will be dedicated towards…
The understanding of rare diseases is often lacking, making it difficult for rare disease patients to get the diagnosis and treatment that they need. Dr. Tracy M. French aims to…
A few years ago, Kyan Harris started experiencing some unwelcome health issues. At just nine years old, the boy wanted to play rugby with his friends. But instead, he was…
According to a recent press release published by the National Institutes of Health (NIH), a new survey spearheaded by the Rare Diseases Clinical Research Network (RDCRN) is seeking to understand…
To read part 1 click here Kristie’s medical mystery was now solved. What was the next step? After speaking with her genetic counselor about her results, Kristie was referred to…
COVID-19 diagnoses are growing at a rapid rate, to the point where data is changing daily. As of today, there are 4.23 million diagnosed cases worldwide, with 1.39 million in…
As reported in WMS, Kathy Miska was beginning to have difficulty walking. This time, she knew what was happening. She'd seen the same thing happen to her sister, who went…
In the face of COVID-19, considerations and procedures regarding the use of radiation for blood cancer patients may need to be modified. When the risk of severe infections from COVID-19…
As reported in Dravet Syndrome News, in a new study, researchers created an artificial intelligence system which could diagnose epileptic conditions more rapidly and easily than existing systems. Epilepsy is…
As reported in Biospace, Adverum Biotechnologies, a gene therapy company focused on clinical stage trials for rare and ocular diseases with unmet needs, has successfully dosed a patient in their…
A 6-year-old girl presented with facial abnormalities, developmental delays, and issues with her renal system. Her family took her to their family physician. Then, when that uncovered no cause,…
By Natalie Homan from In The Cloud Copy The United States goes through blood shortages from time to time when donation numbers drop, usually because of summer travel. In March…
Protalix BioTherapeutics has recently announced the results from the third phase of the BRIDGE trial, which evaluated pegunigalsidase alfa for the treatment of Fabry disease. As these results were positive,…
As the world grows more technologically advanced, so does the medical field. Doctor-patient communication can happen over the phone or email. Telemedicine abounds. More technology is being pointed towards remote…
© Copyright 2024 Patient Worthy
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.
