For patients who lose their vision, artificial and implantable devices like retinal prostheses offer a way forward. But while these prostheses are already somewhat effective, a Korean research team recently…
If you had told me in March, when the viral pandemic began, that we'd still be staying inside 5 months later, I wouldn't believe you. Yet COVID-19 has become one…
According to the Parkinson’s News Today; in the UK, NRG Therapeutics has been awarded £1 million (about $1.3 million) to continue advancing their studies on the mitochondria responsible for producing…
An Australian royal commission is investigating claims that children with disabilities are being excluded from online learning, according to The Guardian. They are also looking into issues with the services…
The winning entry for the 2019 Student Voice was published recently in the Orphanet Journal of Rare Diseases. The prize-winning article, a warm and personal account, was written by Annie…
Happy Friday! We're about halfway through Stevens-Johnson Syndrome Awareness Month. It's a type of severe skin reaction that often involves admission to a burn unit for treatment. Looking for ways…
by Caitlin Seida from In The Cloud Copy As if rheumatoid arthritis (RA) weren’t painful enough, those living with the disorder also have to pay extra attention to their hearts…
According to biotechnology Gilead Sciences, the FDA rejected a new drug application (NDA) for filgotinib. The drug, designed to treat patients with moderate to severe rheumatoid arthritis (RA), is known…
Kara LeFrance Kara LeFrance is diagnosed with acromegaly, a rare disease, and she's been battling it since she was 20 years old. Unfortunately, it took eight years after her first…
According to Parkinson's News Today, diet is something that should be monitored in Parkinson's disease patients. Many do not eat enough iron, vitamin B1, zinc, or vitamin D, which was…
According to the World Health Organization (WHO), there were 228 million cases of malaria across the globe in 2018. Many of these were in African nations; in fact, the African…
Female mammals have XX chromosomes and males have XY. But scientists have never understood how females decide which of their two X's should be active in each of its cells.…
A study conducted at the University of Texas has discovered that combination therapy may be a viable treatment for cholangiocarcinoma with BRAF mutations. Researchers treated participants with both dabrafenib and…
Dr. Harvey Moldofsky Harvey Moldofsky is a professor emeritus at the University of Toronto. His area of focus is SARS, and since the onset of COVID-19, he has been fully…
In a story reported in News Medical Life Sciences, researchers attempting to treat rare diseases often find themselves with a dilemma- while there aren’t many patients to test treatments on in…
Sometimes, researchers find beneficial medications during clinical trials. Other times, the therapy just falls short. According to Myasthenia Gravis News, the latter is true for Firdapse (amifampridine phosphate). The…
This past week, biopharmaceutical company Mustang Bio announced that their gene therapy candidate MB-107 received Rare Pediatric Disease Designation. The gene therapy candidate is designed to treat X-linked severe…
by Lauren Taylor from In The Cloud Copy Myasthenia gravis is a neuromuscular disorder characterized by rapid fatigue and weakness of any muscles that are under the voluntary control of…
To capture the essence of palliative care, the World Health Organization defines it as the prevention and relief of suffering associated with a life-threatening illness. An article in Parkinson’s…
Did you know that over 25 million people worldwide take antidepressants? But even with this high usage, many researchers aren't sure exactly how antidepressants like selective serotonin reuptake inhibitors…
Ross Douthat, a writer for the New York Times, was first diagnosed with Lyme disease and a Bartonella infection in 2015 from a deer tick bite. The effects have lingered…
I-Mab, a biopharmaceutical firm, has begun dosing participants in Phase Ib of their trial of plonmarlimab, a rheumatoid arthritis (RA) treatment. Developers of the medication believe that it could become…
In many cases, autoimmune disorders like myasthenia gravis are treatable. However, there are some cases of refractory (treatment resistant) myasthenia gravis that presents an unmet patient need. However, researchers…
A press release issued recently by Regeneron Pharmaceuticals heralded the acceptance of a Biologics License Application by the FDA for Priority Review of its investigational drug, evinacumab. The Priority…
Ascendis Pharma has just announced that their investigative therapy for achondroplasia called TransCon CNP has just received Orphan Drug Designation from the European Commission. This same designation has already been…
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.
