Elloise Charles has set out on a mission to raise awareness and money for progressive supranuclear palsy (PSP) after her father was diagnosed. His journey to diagnosis was similar to…
A 31-year-old woman from Kota, Rajasthan has recently undergone a surgery performed partially by a robot, according to The Tribune. The intention of the surgery was to remove two tumors…
Victoria and Zack Rasberry are asking for a miracle to save their youngest child, Ollie. Ollie and his older sister, Addy, both have metachromatic leukodystrophy (MLD), a rare disorder that…
The aHUS Alliance has taken on a new project to raise awareness for atypical hemolytic uremic syndrome (aHUS) and the needs of the patient community, according to aHUS News. The…
GeneDx, an OPKO Health company, has recently launched several genetic tests. Within this group of tests are repeat expansion analysis for Friedreich ataxia, spinocerebellar ataxia (SCA), and other forms of…
Charlie Fry is a three-year-old from Kansas City living with SLC6A1. His family is very active in the SLC6A1 community, even starting a nonprofit titled "A Cure for Charlie." They…
The China Center for Drug Evaluation (CDE) has recently granted Harbour BioMed the Breakthrough Therapy designation for batoclimab, a treatment for adults with myasthenia gravis (MG). This designation was created…
Monogram Health has recently announced that they will be expanding their collaboration with Humana Inc. to provide greater access to chronic kidney disease and end-stage renal disease treatment. They will…
There are thousands of rare diseases, making it difficult to remember, much less understand them all. Patient Worthy is here to help, explaining and raising awareness for rare conditions. Today…
Soleno Therapeutics has announced that they will be hosting a Key Opinion Leader (KOL) webinar on Thursday, February 4 at noon EST. This webinar will focus on Prader-Willi syndrome, specifically…
Shane James has recently completed a 24-day long run to fundraise for the Stiff Person Syndrome Research Foundation, earning about $20,000 by the end of his trek. The run, called…
The UK has recently began the practice of the new UK Rare Disease Framework, a government effort that aims to spread awareness, quicken the diagnostic process, and improve treatment and…
One of the biggest concerns associated with the COVID-19 vaccines has been their effect on rare disease patients. There has been minimal research into this topic, leading many to be…
A new licensing and supply deal has been made between Mitsubishi Tanabe Pharma America Inc. (MTPA) and Aquestive Therapeutics concerning the commercialization of riluzole in the United States. Riluzole, being…
Motor symptoms are not the only effects of Parkinson's disease; many affected individuals experience issues with sleep, among other symptoms. In fact, a panel of experts recently came together to…
Kuur Therapeutics has recently released interim data on their CAR-NKT cell therapy platform, which was created for the treatment of hematological and solid malignancies. This data comes from two phase…
The National Institute for Health and Clinical Excellence (NICE), an agency under the UK's National Health Service (NHS), has recently rejected Zeposia. Zeposia is an oral, multiple sclerosis (MS) treatment…
According to an article published in Medscape, guidelines for the treatment of valvular heart disease have changed. The American College of Cardiology (ACC), along with the American Heart Association (AHA),…
Medical professionals have known for a long time that peripheral nerve cell damage plays a major role in Charcot-Marie-Tooth (CMT) disease. A recent study published in Human Molecular Genetics has suggested that…
Parent Project Muscular Dystrophy's (PPMD) goal is to end Duchenne muscular dystrophy (DMD), and they've just made another step in the right direction. They have recently announced the launch of…
Patient input is extremely important when evaluating treatments for rare diseases; Charcot-Marie-Tooth disease (CMT) is no exception. In order to understand the impact of different CMT therapies, researchers from the…
Translate Bio has been moving forward with the development of their new drug candidate, MRT5005. This mRNA therapy was created to address the underlying cause of cystic fibrosis (CF), and…
According to an article in BioSpace, Dyne Therapeutics has recently announced data from preclinical trials of its FORCE platform. This program is aimed at creating treatments for myotonic dystrophy type…
Turkish citizens are putting pressure on their government in regards to access to rare disease treatments, specifically spinal muscular atrophy (SMA) therapies. There are currently two drugs available to Turkish…
Landon Groves was diagnosed with Kabuki syndrome, a rare multisystem disorder, and leukemia soon after his birth. Because of these conditions, his family faces hefty medical bills. His community has…
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