Zachary Thomas has been an advocate for the mucopolysaccharidosis type I (MPS I) community since he was born. Newborn screening is a public health initiative that tests newborn babies for…
Professionals from all corners of the world convene at SSIEM each year to discuss the latest groundbreaking discoveries. According to a report in BioSpace, one such breakthrough was presented by…
Drug development is a long, expensive, and difficult process. Not every treatment being created makes it to the end of the clinical trial process, much less through FDA approval. Takeda…
According to an article published in Yahoo, the FDA has recently granted the Rare Pediatric Disease designation to AVR-RD-05, a gene therapy for Hunter syndrome. This designation is reserved for…
In the European Union (EU), Orphan Drug designation ("Orphan designation") is granted to products intended to treat, prevent, or diagnose a life-threatening, chronic, or rare disease. These are defined as…
When I was a teenager, my mom tried to convince me to become a nurse. As a nurse herself, she told me about the rewards of helping people, the challenges…
ANGEL AID CARES began as the brainchild of Cristol Barrett O’Loughlin, out of a shared desire to pay tribute to her own family experience and to uplift others in need.…
From May 11-14, 2021, the American Society of Gene and Cell Therapy (ASGCT) held its 24th Annual Meeting. Due to COVID-19, the meeting was held virtually - but that didn't…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Fast track designation is granted by the FDA. If a drug developer receives this designation, they begin a process to develop and expedite review for drugs or biologics which treat…
According to BioSpace, Homology Medicines has released the first round of data from the clinical trials of HMI-203, a gene therapy being developed for the treatment of Hunter syndrome, also…
12-year-old Aidan Carter has Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), a rare, progressive condition that impacts mental development, appearance, physical abilities, and organ function. In order…
According to a story from Markets Insider, the biotechnology company REGENXBIO Inc. has recently released data from its phase 1/2 clinical trial. This is interim data from cohorts 1 and…
In late October 2020, Homology Medicines ("Homology") announced its development of a new gene therapy program to treat patients with MPS II, or Hunter syndrome. Homology's mission is to meet…
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 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 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…
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 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…
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