The majority of Parkinson’s patients treated with Levodopa experience relief from the major motor effects of Parkinson’s. However, according to a recent article in Biospace, after prolonged use of Levodopa…
According to a recent article in Fierce Biotech, most cancers progress through constant cell division. Scientists at Vanderbilt University are determined to find the reason for this mysterious cell division.…
According to a story from Charcot-Marie-Tooth News, a recent study has demonstrated the potential impacts of an RNA-based therapy as a treatment for type 1A Charcot-Marie-Tooth disease. The treatment was…
Newron Pharmaceuticals and Zambon have entered into an agreement that has the potential to produce a label extension for a treatment for Parkinson's. The agreement is focused on safinamide as…
The Patient-Centered Outcomes Research Institute (PCORI) has announced that it will be contributing funding for four new studies focused on four different rare diseases. The funding will top out at…
Genetec Engineering and Biotechnology News recently carried a story giving credit to Baylor College researchers for finding microbes living in the gut that are associated with specific symptoms of neurological…
We seldom see articles about multiple system atrophy (MSA also called Shy-Drager syndrome). The disease is extremely rare and currently, there is no effective treatment. But according to a recent…
Science 37 and Xperiome have entered into a partnership in order to allow for more viable rare disease research, according to an article in PR Newswire. If the partnership goes…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Patients with hereditary angioedema (HAE) experience recurrent "attacks," or periods of swelling under the skin. Now, in a Phase 2 clinical trial, researchers are evaluating PHVS416 for patients with…
A report, commissioned by the National Organization for Rare Disorders and released by IQVIA, has revealed that the majority of orphan drugs, 79%, treat only rare diseases. Despite this fact,…
It is estimated that one person in forty to sixty thousand is affected by Fabry disease. Approximately five hundred Canadians are known to have Fabry disease. Newswise recently carried the…
According to a study from Hindawi, a team of scientists have reported the discovery of never before seen mutations that are linked to Alport syndrome, a rare kidney disease. These…
Pharnext has released an update on the clinical development of PXT3003, a treatment for Charcot-Marie-Tooth disease type 1A (CMT1A). According to the biopharmaceutical company, they are on track to dose…
A recent article in the journal Nature describes the first draft of human genome analysis and sequencing published in 2001 by the Human Genome Project international consortium. The consortium included…
When a patient is told that they have a life-threatening rare disease and then told that there is no cure, every effort is made to attend to their physical needs.…
The Orange County Guide features doctors’ articles in each issue. Dr. Justin West’s search to cure his son’s rare disease will be familiar to families also in search of…
Vanderbilt University has just announced a new collaborative agreement with Soleno Therapeutics in aim to develop KATP channel activators as a therapeutic for rare diseases. This therapy has already been…
As reported in News Wise, these oncologists don’t like their options. They want to provide patients with the best possible options to tackle the lethal cancer mesothelioma, but their hands…
A recent study has documented the differences between two rare forms of childhood brain inflammation. The two similarly presenting conditions are often confused for one another. A better understanding of…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to BioSpace, Kronos Bio will continue with its plans for a Phase 3 trial of entospletinib after a positive meeting with the FDA. One of the meeting's focuses was…
A recent study published in Cell Reports has uncovered a mechanism where germs living within the lungs of cystic fibrosis (CF) patients produce a type of carbohydrate slime that minimizes the efficacy…
James Dahlman, a scientist and professor at both Emory University and the Georgia Institute of Technology, has partnered with the CMT Research Foundation. The aim of this partnership is to…
A new study published in JAMA Oncology has found that of all patients who have had cancer spread to their bones and started treatment, 2,8% have developed osteonecrosis within their jaw, also…
© 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.
