Chronic kidney disease patients often develop aortic stenosis, which requires a transcatheter aortic valve replacement (TAVR) procedure to treat. The question arises: will a TAVR procedure worsen the renal function…
Dane Tidwell lost his leg to osteomyelitis when he was just 18 years old. He spoke about the confusion and loneliness that came with being an amputee, and how his…
Sarepta Therapeutics has met with the Office of Tissues and Advanced Therapies of the FDA in order to discuss a Type C written response about a clinical trial for SRP-9001,…
According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…
Mononine, a treatment for hemophilia B, hit the market nearly three decades ago in 1992. Since then, there have been amazing advancements in the knowledge and treatment for this disease.…
There is currently an unmet medical need for therapies and research for Parkinson's disease, with the incidence increasing drastically. In fact, the number of cases around the world is expected…
The SLEEP 2020 Annual Meeting will be held virtually due to the COVID-19 pandemic, but that is not stopping Axsome Therapeutics from presenting the results of their Phase 2 CONCERT…
Cornell University and reVision Therapeutics have come together through a licensing agreement to develop a treatment for Stargardt disease, according to BioSpace. They will be working on and commercializing Cornell's…
Rocket Pharmaceuticals has recently dosed the first patient in their trial of RP-A501, a treatment for Danon disease. According to BioSpace, this trial will test the safety, tolerability, and efficacy…
Research was presented at The Digital International Liver Congress that proved nidufexor is a safe and viable treatment for nonalcoholic steatohepatitis (NASH), according to Healio. It was shown to reduce body…
According to Charcot-Marie-Tooth News, the EveryLife Foundation has began a scholarship fund to help rare disease patients in the United States. Living with a rare disease often brings challenges, many…
Health Canada has recently granted market authorization to Hoffman La-Roche Limited for their neuromyelitis optica spectrum disorder treatment, ENSPRYNG. Canada is the first country to offer this medication, and hopefully…
This month is Charcot-Marie-Tooth (CMT) Awareness Month. Due to COVID-19, there cannot be any of the typical events, but that doesn't mean there's nothing you can do. From a virtual…
According to BioSpace, a Phase III trial of Farxiga has produced very positive results. This chronic kidney disease (CKD) treatment was able to dramatically prolong survival in those with stages…
Dealing with grief is different for everybody. Unfortunately, living with a rare disease can bring sadness and grief into one's life. That is why the Livestrong Foundation has offered a…
A study has concluded that Neupro Patches are a viable treatment option for those living with Parkinson's disease, according to a Parkinson's News Today article. The patch, which can treat…
XPhyto Therapeutics has been developing a skin patch as a delivery system to treat Parkinson's disease. They just released an update, stating that the "formula was locked," and the process…
According to an article in Newswire, medical professionals from the UK have discovered a number of protein structures that are associated with Parkinson's disease. The hope is that further research…
According to an article from Morningology, the pharmaceutical company Teva has recently been charged by the U.S. Justice Department for conspiring to raise generic drug prices. This is a serious…
The CMT Research Foundation has announced that September will be Charcot-Marie-Tooth (CMT) Disease Action Month. The switch from awareness to action illustrates just how much CMT patients and their families…
According to an article from Newswire, the Alzheimer's Association is awarding Elixiron Immunotherapeutics one million dollars for work on their Alzheimer's treatment, EI1071. This money will go towards a clinical…
New Zealand has the third highest rate of inflammatory bowel disease (IBD) in the world, but there are only two drugs that are currently being funded to treat it. As…
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…
The importance of potassium restrictions in the diets of chronic kidney disease (CKD) patients has been stressed for years. It lowers levels of serum potassium, therefore lowering the mortality risk.…
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,…
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