This is my son Nikolai. Nikolai was recently diagnosed with Mucopolysaccharidosis type 2, or "Hunter Syndrome." A rare genetic form of childhood dementia, it's caused by a mutation in the…
The pharmaceutical industry celebrates a significant victory for rare disease patients as the Food and Drug Administration approved Denali Therapeutics' groundbreaking enzyme replacement therapy for Hunter syndrome, marking a pivotal…
As reported on FierceBioTech, the U.S. Food and Drug Administration has released its complete response letter (CRL) detailing why it declined to approve Regenxbio’s experimental gene therapy, RGX‑121, for Hunter…
REGENXBIO encountered a significant regulatory obstacle when the FDA issued a complete response letter regarding its biologics license application for RGX-121, an investigational gene therapy targeting mucopolysaccharidosis II (MPS II),…
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…
A newly published Phase 1/2 study in The New England Journal of Medicine, and reported on by the Manila Times, highlights promising early results for tividenofusp alfa (DNL310), Denali Therapeutics’ investigational…
As discussed in a recent interview between Krystal Jacques and Prof. Brian Bigger, a novel stem cell–based gene therapy is showing early promise as a potential one-time, lifelong treatment for…
As reported by NewsWise, new clinical study led by investigators at the University of North Carolina at Chapel Hill is offering rare hope for families affected by Hunter syndrome, a…
Denali Therapeutics (Nasdaq: DNLI) announced that the U.S. Food and Drug Administration (FDA) has extended the review period for its Biologics License Application (BLA) for tividenofusp alfa, an investigational therapy…
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…
Diagnosing Rare Diseases Diagnosing rare diseases is historically difficult. Physicians are taught to think about horses instead of zebras because horses are abundantly more common. For instance, if you're tired…
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 study published in the journal Blood Advances has documented that hematopoietic stem cell transplantation (HSCT) is both safe and effective for children with many different kinds of inherited nonmalignant conditions…
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