CohBar has initiated dosing in their Phase 1b trial of CB4211, which is a treatment for nonalcoholic steatohepatitis (NASH). The biotechnology company focuses on mitochondria based therapies with the goal…
Professor Illana Gozes and her team at Tel Aviv University recently developed NAP (CP201). It is an experimental drug that has been proven to be effective in the treatment of…
According to a story from Charcot-Marie-Tooth News, the nonprofit organization CMT Australia, which focuses on serving the Charcot-Marie-Tooth disease patient community in Australia, has recently acquired a grant to the…
The Michael J. Foxx Foundation (MJFF) has recently award $8.8 million of grants to various medical professionals for their research on Parkinson's disease. The aim is to develop more treatments,…
According to a story from england.nhs.uk, a recent agreement struck between the UK's NHS and Vertex Pharmaceuticals will herald a major improvement for the many of the country's cystic fibrosis…
By Lauren Taylor from In The Cloud Copy Onconova Therapeutics, Inc. is a biopharmaceutical company that works to develop new drugs to treat cancer, primarily focusing on myelodysplastic syndromes. Myelodysplastic…
by Danielle Bradshaw from In The Cloud Copy It has been found that fingolimod - a drug typically used for multiple sclerosis - may also be effective in treating ANCA-associated…
By Lauren Taylor from In The Cloud Copy Myasthenia gravis is an autoimmune condition characterized by muscle weakness and fatigue of muscles that are under the patient’s voluntary control. This…
Salk Institute scientists have scored a significant gain in their quest to find a functional treatment for type 1 diabetes. According to an article in Salk News, scientists at the…
Of the things we take for granted about our existence, I venture to say vision is pretty high on the list. Even if your eyesight starts to go it can…
Editas Medicine has developed a sickle cell disease (SCD) treatment, and it has recently received the Rare Pediatric Disease designation from the FDA. This means that their therapy, EDIT-301, is…
According to a recent article in FierceBio, Andrea Mazzocchi, Known Medicine CEO, is not trying to reinvent the wheel. On the contrary, her company will be providing a tool to…
It goes without saying that change is not easy for most people. If life has been a certain way for many years and something alters the course of things, it…
According to an article in the publication Labroots, autism spectrum disorder (ASD) may be the result of interaction between environmental and genetic factors. For instance, researchers point out that ASD…
In 2012, a new medical technology emerged: CRISPR-Cas9, which offered the opportunity for gene editing. LabBiotech explains that gene editing could benefit patients with genetic disorders by adding, deleting, or…
In a recent press release, biopharmaceutical company Aligos Therapeutics announced that the first patient was dosed in their Phase 1a/b clinical trial. The trial is designed to assess ALG-010133 as…
According to MedCity News, a third patient dosed with AT132 during the ASPIRO trial has died. The Phase 1/2 clinical trial tested the efficacy, safety, and tolerability of AT132 for…
Huda Zoghni was born in Lebanon. Huda attended medical school at the American University located in Beirut in 1975. According to an article in Discover, Huda was forced to leave…
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…
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…
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…
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…
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…
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…
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