As reported on drugs.com, REGENXBIO has disclosed that the U.S. Food and Drug Administration has halted clinical testing of its investigational gene therapies RGX‑111 and RGX‑121, both being developed for…
As reported on Drugs.com, REGENXBIO has announced that the U.S. Food and Drug Administration (FDA) has halted clinical testing of its investigational gene therapies RGX‑111 and RGX‑121, two programs targeting…
Newborn screening is an important public health initiative that checks for rare and serious genetic, blood, hormonal, or metabolic conditions at birth. The test collects blood through a heel…
617 words (source - 3% match) vs. 452 words (mine - 4% match) As our healthcare field continues to innovate and grow, we have seen more conversations regarding gene therapy…
Right now, there are only an estimated 10 people in Ireland who have been diagnosed with a form of mucopolysaccharidosis type I (MPS I) called Hurler syndrome. This is the…
On August 25th, 2022, the American Society of Gene & Cell Therapy held a webinar as part of its Lunch and Learn series, which is intended to help people living…
According to a recent article, the family of a young boy from Australia who is diagnosed with the rare genetic disease mucopolysacchariodosis type 1 (MPS 1) is sharing his story to…
Currently, the standards-of-care for patients with Hurler syndrome, or one of the forms of mucopolysacchardiosis type I (MPS I), include enzyme replacement therapy and hematopoietic stem cell transplants. However, Medical…
At the very end of March 2021, specialty pharmaceuticals company JCR Pharmaceuticals Co., Ltd. ("JCR") shared that its investigational drug candidate JR-171 received Orphan Drug designation from the European Medicines…
According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…
India first drafted a national rare disease policy back in 2017. Unfortunately, it was never enacted due to budgeting and implementation issues. This draft included suggestions such as a corpus…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Orchard Therapeutics recently announced the release of some data from an ongoing proof-of-concept study. This special trial is testing the company's…
Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…
The Daily News recently published an announcement by researchers at Sangamo Therapeutics showing the results of its first human trial to treat two rare genetic disorders through gene-editing technology. Gene-editing (or…
Gene Editing What is gene editing? It's been a hot topic over the last few years, but basically the concept is this: instead of disabling a malfunctioning gene, researchers are…
According to a story from medicalxpress.com, a team of scientists at the University of Massachusetts Medical School have successfully developed a strategy for editing genes that could be used to…
The first patient has been treated in the Empowers study, a clinical trial investigating an experimental gene editing therapy for the treatment of mucopolysaccharidosis type I. The source article, which…
The 15th International Symposium on MPS and Related Diseases An event geared towards both medical experts and families affected by MPS diseases such as Chudley Rozdilsky syndrome and Hurler syndrome,…
The Virginia Newborn Screening Advisory Committee met late last week to determine the fate of Pompe Disease and MPS-1 screening in VA. Before the vote took place, some families spoke…
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