According to a story from PR Newswire, the biotechnology company REGENXBIO Inc. has recently announced plans to expand its RGX-121 developmental program. RGX-121 is being developed as a potential one-time…
A recent meta-analyses of clinical studies and case reports that examined the safety of idursulfase therapy, which is an enzyme therapy, has shown that this form of treatment is…
East Anglia's Children's Hospices in Norfolk, U.K., has recently opened up its new facilities after renovations. To celebrate this opening, Kate Middleton, the Duchess of Cambridge, visited and spoke…
According to a story from BioSpace, the biopharmaceutical company CANBridge Pharmaceuticals announced that it has recently submitted a New Drug Application with China's National Medicinal Products Administration (NMPA) for its…
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 globenewswire.com, the biopharmaceutical company Denali Therapeutics, Inc. has recently announced that its experimental product candidate DNL310, which is currently being developed as a treatment for…
According to a story from Palomar Health, a patient with Hunter syndrome is about to finish up the company's Pathmaker Internship program. The patient in question is Kyle Underwood, who…
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 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 Pharmaphorum, there are a number of new treatments that have been introduced or have seen major strides in development over the last year. More patient…
According to a report by CheckOrphan, a recent study investigated the use of genome editing directly in the body. The research involved the use of these editing techniques in cases…
According to a story from the Santa Cruz Sentinel, the earliest results from a critical gene editing study could offer some encouragement for those hoping to treat rare genetic diseases…
According to a story from philly.com, four year old Ethan Lieber of the Roxborough neighborhood in Philadelphia is one of just 500 people in the US with Hunter syndrome. This…
According to a story from The Tennessean, mother Melissa Hogan has been inspired by her son Case's Hunter syndrome to start a nonprofit organization called Project Alive. The primary purpose…
Creative parenting. I find such inspiration when I hear about parents digging deep to adapt surroundings (or life) so their kids can deal with it better. A WBTV social media…
Easton was born at thirty-six weeks gestation on January 6, 2017. He was almost immediately taken to the NICU for something called PPHN (Persistent Pulmomary Hypertension), Easton was intubated for…
Today, Professor Elsa Shapiro and Dr. Christina Lampe presented "Cognitive Impairment in Patients with MPS II: From Disease Burden to Cognitive Testing" at Satellite Symposium, an event sponsored by Shire…
Gene therapy is on the rise, and it just might be exactly what many children suffering from genetic disorders need to save their lives. Today reported that an amazing organization…
REGENXBIO announced last month that the Investigational New Drug application (IND) is active for a Phase I/II clinical trial of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II)…
REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they're developing,…
Sangamo Therapeutics shared some big news about the search for Mucopolysaccharidosis II (MPS II) treatment. They reported that they have, for the very first time, used experimental in vivo gene therapy to treat…
Sean Kisielnicki's life is ruled by Hunter syndrome. This rare condition not only affects him, but takes a toll on the entire family. The morning routine is always the same.…
Karl Swaine is nine years old. He has a rare progressive and genetic disorder called Hunter syndrome. This disorder prevents the body from breaking sugar molecules down. Throughout the world,…
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