The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…
Females vs. Males with Fabry Fabry disease is an X-linked condition caused by a malfunctioning GLA gene. It was long believed to only affect men. Since women have two X…
According to a story from Euroinvestor, the drug development company Resverlogix Corp. recently announced that it has received the necessary $2.9 million in funding for a Phase 2 clinical trial…
Fabry Disease Fabry Disease is a rare lysosomal storage condition. Mutations in the GLA gene cause a type of fat called globotriaosylceramide to build up in the body's cells. Symptoms…
The State of Rare Disease in India There are 7,000 known rare diseases worldwide. The Organization for Rare Diseases India (ORDI) estimates 70 million people are affected by a rare diagnosis…
The typical treatment for Fabry disease is Fabrazyme which is an enzyme replacement therapy (ERT). This therapy must be administered to the patient every two weeks. A new ERT created by…
According to a story from Fabry Disease News, the drug developer Avrobio recently announced that the US Food and Drug Administration (FDA) has given the company's experimental drug AVR-RD-01 Orphan…
Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…
According to a story from globenewswire.com, the gene therapy company AVROBIO recently announced that the company's experimental gene therapy drug candidate AVR-RD-01 has earned Orphan Drug designation from the US…
We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…
According to a story from The Sydney Morning Herald, the Australian government recently announced that it will be adding Galafold, a drug used to treat rare Fabry disease, to the…
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…
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