According to a story from Fabry Disease News, the first patient in a clinical trial testing the investigational gene therapy FLT190 has reportedly been dosed. FLT190 is being developed as…
According to a publication from Fabry Disease News, Amicus Therapeutics has as many as 14 investigational drugs currently in development for the treatment of rare diseases including Fabry, Pompe, and…
A new study conducted by researchers in Japan has uncovered a new potential way to diagnose Fabry disease. Fabry Disease Fabry disease is an illness which inhibits the patient from producing the…
According to a story from globenewswire.com, the drug company Amicus Therapeutics recently announced that Argentina's Agency of Medicines, Food, and Medical Devices (ANMAT) has granted approval for the drug migalastat…
According to a story from Korea Biomedical Review, two Korean drama television shows, Partners for Justice Season 2 and Doctor John have recently depicted Fabry disease as key components during their run, which…
According to a publication from Fabry Disease News, biotechnology company Avrobio recently announced that early phase 1 and 2 clinical studies of experimental Fabry treatment AVR-RD-01 have yielded encouraging results.…
According to a story from health.economictimes.indiatimes.com, the country of India is currently beginning to take more steps in order to address the dire unmet medical need of its rare 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…
According to a story from Fabry Disease News, a recent study has found that despite changes to sperm cells, the fertility of men with Fabry disease is not affected. The…
According to a press release from the Massachusetts-based biotechnology company Avrobio, Inc., clinical tests of the Company's experimental Fabry disease gene therapy, AVR-RD-01, have yielded impressive preliminary results. Though the…
According to a publication from Fabry Disease News, Russian researchers recently screened over five-and-a-half thousand patients for Fabry disease while they continued with prescribed hemodialysis. The researchers' study, published in…
Extended Collaboration Amicus Therapeutics has just announced that they are expanding their collaboration with the University of Pennsylvania's Perelman School of Medicine for the next five years. This collaboration is…
According to a publication from Fabry Disease News, the U.S. Food and Drug Administration (FDA) has approved American biotechnology company Avrobio's Investigational New Drug (IND) application for experimental Fabry disease…
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…
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