In a recent statement, Scott Gottlieb, the Commissioner of the US Food and Drug Administration, has re-affirmed the organisation’s commitment to modernising drug regulatory pathways. New changes are hoped to…
According to a story from Pharma Voice, Dr. Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA) has recently released a statement in regards to efforts…
According to a story from PR Newswire, a new program called The Lantern Project is working to provide a free testing program that allow doctors to more quickly diagnose patients…
Avrobio has released updates on patients in two clinical trials of a potential gene therapy for Fabry disease. According to the announcement, two patients taking part in a Phase 1…
The Malaysia Lysosomal Diseases Association (MLDA) was formed in 2011 by the hands of eight families. Their goal was to raise awareness and funds for those diagnosed with lysosomal storage…
According to a story from CheckOrphan, Durhane Wong-Rieger, the CEO of the Canadian Organization for Rare Disorders, says that the Canadian health system has been letting down the roughly three…
According to a story from MD Magazine, the US Food and Drug Administration (FDA) has approved the medication migalastat, also known as Galafold, for the treatment of Fabry disease. This…
According to WBUR, treatments like CAR-T and CRISPR are on the cutting edge. They’re important to keep an eye on as the future of medical technology and treatment. They’re not…
So what is Fabry disease you ask? You came to the right place! Fabry disease is a genetic, lysosomal storage disorder that occurs when a specific kind of fat known…
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…
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