During the National Institutes of Health (NIH) Rare Disease Day at NIH event, a panel was held titled "From Kitchen Tables to Changing Paradigms — Advocacy as a Driving Force…
A recent study has illuminated the need for further research into how different forms of exercise may provide certain benefits for patients living with Charcot-Marie-Tooth disease (CMT). This study was…
Sarcoma Sarcoma is a cancer that accounts for 1% of all adult cancers. It can affect individuals of any age and can impact any part of the body. It grows…
The FDA has just announced their approval of Evkeeza as a therapy for those living with homozygous familial hypercholesterolemia. The therapy is owned by Regeneron Pharmaceuticals. It was evaluated with…
Pharnext has announced that they will soon open enrollment for their PREMIER trial of PXT3003, a Charcot-Marie-Tooth disease type 1A (CMT1A) treatment. Beginning in March, researchers will search for 350…
Researchers from the Universities of Nottingham and Exeter have discovered a new, possible treatment method for Duchenne muscular dystrophy (DMD). Their research demonstrated that treating hydrogen sulfide (H2S) deficiency in…
In a recent study, researchers have uncovered a second gene located within the mitochondria that is associated with Charcot-Marie-Tooth Disease (CMT). This study was recently published in Annals of Neurology. CMT…
A woman living with Charcot-Marie-Tooth Disease type 2 (CMT2) has experienced symptom improvement following therapy with an anti-inflammatory compound found in green tea. This study was recently published in Medicina.…
According to GlobeNewswire, Tiziana Life Sciences and Parexel Biotech have agreed to collaborate on a Phase 1b/2 trial of foralumab, an oral treatment for Crohn's disease. As previous research has…
Lysogene has just announced that the FDA has approved their Investigational New Drug Application (IND) for a therapy for GM1 gangliosidosis, a rare pediatric condition. The investigational treatment is a…
Julia's Wings Foundation is gearing up for the Operation Wear Red Campaign in an effort to raise awareness for the rare blood disorder aplastic anemia. From the first of March…
During a recent WZDX News interview with Dr. David Bick, HudsonAlpha Faculty Investigator Dr. Bick stated that in Alabama alone, there are approximately four hundred thousand people with rare diseases.…
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, Biohaven Pharma has utilized their MATE platform to create and develop a hyperimmune globulin mimic (HGM) that is able to both bind to and neutralize various strains…
Billions of dollars have been spent in research over decades, resulting in only two therapies that treat symptoms of Alzheimer’s disease (AD). There are no approved methods that diminish the…
According to a story from epmmagazine.com, a team of researchers from the Universities of Exeter and Nottingham have recently completely a study that could lead to the development of a…
The Cystic Fibrosis Foundation is dedicated to finding a cure for the rare, genetic disorder. They fund more CF research than any other organization, and their latest effort is a…
According to a story from One News Page, the biopharmaceutical company BridgeBio Pharma and its affiliate ML Bio Solutions have recently announced that dosing has begun in its phase 2…
The FDA has recently accepted NeoImmuneTech's Investigational New Drug (IND) application for NT-17, a treatment for progressive multifocal leukoencephalopathy (PML). Now that the application has been cleared, the biopharmaceutical company…
As reported in Biospace, Taysha Gene Therapies wants to eradicate devastating genetic epilepsies and central nervous system disorders rooted in a single genetic mutation. That’s going to take some innovative…
According to a story from businesswire.com, the biopharmaceutical company Ipsen recently presented a total of 9 study abstracts at the recent 18th annual conference of the European Neuroendocrine Tumor Society…
From February 25-27, the Americas Committee for Treatment and Research in Multiple Sclerosis will run its ACRTIMS Forum 2021, according to BioSpace. At this forum, Clene Inc. will present interim…
A Phase 3 study of upadacitinib, an ulcerative colitis treatment, met both its primary and secondary endpoints, according to an announcement from AbbVie. The trial, titled U-ACCOMPLISH, saw a third…
Research by scientists at Cornell University and Weill Cornell centered around the endothelial cells that form a barrier between tissues and vessels. They control the flow of fluid and substances…
As the pandemic rages on, doctors across the country are beginning to notice a trend. A number of children who have contracted COVID-19 have also been impacted by a rare…
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