For patients with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), post-exertional malaise (PEM) can be extremely debilitating and disruptive. PEM occurs when symptoms worsen or become more severe within 12-48 hours…
According to a story from Targeted Oncology, an experimental combination therapy tested on patients with advanced clear cell renal cell carcinoma (ccRCC) appeared to show potential for efficacy in a…
In late September, GlaxoSmithKline announced FDA approval of its treatment, Nucala, for patients with hypereosinophilic syndrome (HES). The treatment is safe for patients older than 12. Additionally, this approval is…
As written in BioSpace, Genetech has recently presented new two-year data on their spinal muscular atrophy (SMA) treatment, evrysdi. The information was released at the 25th International Annual Congress of…
On September 30, 2020, BioCryst Pharmaceuticals ("BioCryst") announced promising data on their drug candidate BCX9930. The therapy, an orally administered Factor D inhibitor, is designed to treat patients with previously…
The FDA has recently granted two designations, the rare pediatric disease and orphan drug designations, to Spirovant Sciences' gene therapy for cystic fibrosis (CF). SPIRO-2101 will now be able to…
Nate Peters is an air force veteran, and he also lives with ankylosing spondylitis. This means that he knows what it's like to spend a lot of time in the…
Historically, clinical trials have played an immense role in collecting patient data, understanding disease progression, and crafting more targeted treatments. This is especially true in the oncology realm. But according…
Eisai has announced that they will begin a Phase 3 trial, titled MOMENTUM, to evaluate lorcaserin as a treatment for Dravet syndrome. This decision comes after a consultation with the…
The company Lidl GB has just announced a recall for Cologran Stevia tablets. These sweeteners contain aspartame, or E951. Although not harmful for most people, it is essential for this…
A biological marker ("biomarker") is a measurable trait or characteristic that can tell researchers something about your body or health. For example, body mass index (BMI) is a common biomarker…
According to a story from PR Newswire, Chiesi Global Rare Diseases and the biopharmaceutical company Protalix BioTherapeutics, Inc. have recently announced that they have launched an Expanded Access Program (EAP)…
According to a story from BioSpace, the biopharmaceutical company Mereo BioPharma Group recently announced that its investigational therapy setrusumab has earned Rare Pediatric Disease designation from the US Food and…
Since 2012, the nonprofit organization Dysautonomia International has organized Dysautonomia Awareness Month. With the goal of spreading awareness of the condition in the medical community and among the general population,…
Seelos Therapeutics and Duke University have both signed a Sponsored Research Agreement, meaning they will work in tandem to evaluate and test SLS-004, a Parkinson's disease treatment. Their research aims…
October is Rett Syndrome Awareness Month, and even though the pandemic has changed the way that we celebrate it, there are plenty of ways to get involved! Rettsyndrome.org has organized…
According to a story from the AKU Society, the European Medicines Agency (EMA) has recently announced a positive opinion on extending the label indication for nitisinone (marketed as Orfadin®). The…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Let me begin by saying that…
The Value of a Service Dog September has been deemed National Service Dog Month. These animals are essential for many people living with chronic conditions, disabilities, and various rare diseases.…
A study reported in NewsWise evaluates retinoid-x receptor (RXR) agonists as a treatment for recurring and refractory multiple sclerosis (MS). This specific RXR agonist, bexarotene, was found to help remyelination,…
aHUS Tracking Atypical hemolytic uremic syndrome (aHUS) is a rare disease that causes blood clots to form within the small blood vessels in the kidneys. These clots lead to organ damage…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to an article from BioSpace, Vivet Therapeutics and Pfizer have entered into a manufacturing agreement to develop Vivet's gene therapy, which treats Wilson disease. The terms of the agreement…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Dave Kinler and I have walked…
Damian Green from the University of Washington is the Principal Investigator for a new research project in multiple myeloma. This project is thanks to a research agreement between the Fred…
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