Did you know that October is considered Gaucher Awareness Month? Although October is soon coming to an end, we at Patient Worthy want to advocate for continued awareness and…
Six years ago, the United MSD Foundation began its annual Zebra Run with a goal of raising multiple sulfatase deficiency (MSD) awareness, as well as garnering funds to advance…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare or life-threatening illnesses, defined as those affecting under 200,000 people or…
In a recent news release, rare disease-focused biopharmaceutical company CANbridge Pharmaceuticals Inc. ("CANbridge") shared that the National Medical Products Administration (NMPA) of China accepted an Investigational New Drug (IND) application…
On August 2, 2021, biotechnology company Amicus Therapeutics ("Amicus") shared that its oral treatment Galafold (migalastat) was approved by the European Commission (EC) for the treatment of adolescent patients (ages…
According to a recent news release from biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx"), the company's therapy Mepsevii (vestronidase alfa) was approved in Spain for reimbursement. The entire reimbursement process takes…
New research suggests that ultrasounds could be a new diagnostic tool for patients with a rare lysosomal storage disorder. According to Fabry Disease News, kidney dysfunction is characteristic in Fabry…
Over this past week, the Harrington Discovery Institute announced five recipients of its introductory Harrington UK Rare Disease Scholar Award competition. The Institute, which is involved with Cleveland's University Hospitals,…
To determine the safety and efficacy of experimental gene therapy ST-920, researchers first examined the impact of the drug on mouse models of Fabry disease. As reported in Fabry Disease…
According to a press release from Takeda Pharmaceutical Company published by For Press Release, the Company has launched a collection of enzyme replacement therapies for use in the treatment of…
If you had the opportunity to gaze into a crystal ball and see the future, would you do it? And if you did, and you noticed something amiss, would you…
Fabry Disease is the number number two Lysosomal Storage disease in terms of numbers affected. It is an X-linked disease, with males passing on the mutated allele to all of their…
In 1964, Henri Hers was studying Pompe disease when he suggested that the absence of lysosomal enzymes could be responsible for several other conditions. He was right, and a category of…
Maroteaux-Lamy Syndrome, also known as mucopolysaccharidosis (MPSVI), is a lysosomal storage disorder. It's one of a number of disorders that affects specific enzymes in specific locations. The job of these…
What if you had an inherited disorder that caused a fat called globotriaosylceramide to build up in your cells? Chances are pretty good that you'd be diagnosed with Fabry disease,…
Fabry disease results from deficient activity of alpha-galactosidase A (a-GAL A). Bless you. What? That's an enzyme which breaks down complex sugar-lipid molecules called glycolipids. When the body can't break…
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