According to a story from globenewswire.com, the pharmaceutical company Amicus Therapeutics recently announced that its drug Galafold had received official approval for public use in Japan. Galafold is the first…
According to a story from macleans.ca, Julia Alton felt hopeless when she first started to experience the symptoms of her Fabry disease. However, she refused to let the disease hold…
According to an article from globenewswire.com, the pharmaceutical company Amicus Therapeutics announced that the Food and Drug Administration (FDA) has the company's New Drug Application for migalastat with filing for…
New drugs can often make a huge difference in a patient's life. Especially in the case of rare conditions like Fabry disease, where there may be a limited number of…
The pharmaceutical company Protalix Biotherapeutics, Inc. announced that a drug currently in development called PRX-102 has been granted orphan drug status by the European Commission. The treatment is being developed…
The Canadian Fabry Association wants to create awareness for rare diseases and you can help! They want to get as many people to wear a Be Rare Be You tattoo…
Amicus Therapeutics is an American therapeutics company based in Cranbury, New Jersey. They're new groundbreaking treatment announcement, however, has very little to do with America, or even New Jersey for that…
Most people are familiar with the old adage, “The squeaky wheel gets the grease.” My grandmother used to go a bit further by pointing out, “But the greased wheel that…
Earlier this year, Kyle Petty made his way across the country for his annual fundraiser appropriately called "Ride Across America." While Petty, a famous NASCAR racer, says one of his…
If you’re a science nerd, we live in some pretty exciting times. Even though we’re going through a moment where it feels like science and empirical evidence itself is under…
Want some more news, events and announcements on Fabry disease? We got 'em! [one_half] [/one_half] [one_half_last] This Fabry Disease Treatment Has Had a Rocky Journey Regulatory Pathway Announced…
One of my favorite scenes from Forrest Gump was the titular main character running across the United States. He got all kinds of press, as well as masses of people…
Most people have heard the term “rare disease,” but far fewer can name a rare disease let alone imagine what life might be like for those who have one. When…
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…
Happy Pre-Thanksgiving Week Friends! It is almost Thanksgiving which is one of our FAVORITE holidays here at Patient Worthy. Why? Because on this holiday, we are focused on gratitude, family and…
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…
I read a report recently from the New England Journal of Medicine about a clinical trial and thought I’d share it with you because it’s about Fabry disease, a genetic disease…
I’m impressed of with the direction of the Fabry Support & Information Group (FSIG) and the offerings they have. In August 2016, they had a Fabry Family Get Together at…
When you have a rare disease such as Fabry disease, it’s easy to feel alone. As reported on the National Fabry Disease Foundation’s website, no one really knows how many…
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,…
I applaud the American Association of Kidney Patients for their efforts to raise awareness about a condition called Fabry disease. A rather rare genetic disease, Fabry disease seems to affect…
If you've got Fabry Disease and you live in Canada, you've GOT to check this out. If you don't have Fabry Disease, allow me to clue you in: It's a…
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…
Lysosomal Storage Disorder, or LSD, is a catch-all term for more than 45 inherited metabolic disorders that result in the build-up of various toxins in the patient's cells. Currently, there is treatment…
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