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…
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…
An Indian health policy initiative meant to provide funding to rare disease patients has struggled to gain approval from the country's administration, according to a publication from DNA India. First…
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…
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…
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