Jay’s Hunter Syndrome Story
All genetic testing we did for Jay during pregnancy came back normal (though we did the bare minimum). When he was born, he struggled to breathe and was diagnosed with…
Continue Reading
Jay’s Hunter Syndrome Story
Liam’s Hunter Syndrome Story
Editor's Note: This story was originally written by Anne Denneen, and shared on Caring Bridge. So many things happen in life that you don’t even register them at the time. …
Continue Reading
Liam’s Hunter Syndrome Story
Raising Awareness of Hunter Syndrome: Stephanie’s Caregiver Story
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…
Continue Reading
Raising Awareness of Hunter Syndrome: Stephanie’s Caregiver Story
Breaking Through the Rare Disease Barrier: FDA’s Historic Approval of Denali’s Hunter Syndrome Treatment
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…
Continue Reading
Breaking Through the Rare Disease Barrier: FDA’s Historic Approval of Denali’s Hunter Syndrome Treatment
FDA Explains Rejection of Regenxbio’s Hunter Syndrome Gene Therapy
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…
Continue Reading
FDA Explains Rejection of Regenxbio’s Hunter Syndrome Gene Therapy
Gene Therapy Setback: REGENXBIO’s RGX-121 Faces Regulatory Hurdles in Ultra-Rare Disease Treatment
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),…
Continue Reading
Gene Therapy Setback: REGENXBIO’s RGX-121 Faces Regulatory Hurdles in Ultra-Rare Disease Treatment
FDA Places Clinical Holds on REGENXBIO’s MPS Gene Therapy Programs After Tumor Detected in Trial Participant
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…
Continue Reading
FDA Places Clinical Holds on REGENXBIO’s MPS Gene Therapy Programs After Tumor Detected in Trial Participant
FDA Places Clinical Hold on REGENXBIO’s MPS Gene Therapy Programs After Tumor Case Review
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…
Continue Reading
FDA Places Clinical Hold on REGENXBIO’s MPS Gene Therapy Programs After Tumor Case Review
NEJM Publishes Phase 1/2 Results for Denali Therapeutics’ Brain‑Penetrant Hunter Syndrome Therapy
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…
Continue Reading
NEJM Publishes Phase 1/2 Results for Denali Therapeutics’ Brain‑Penetrant Hunter Syndrome Therapy
One-Time Gene Therapy Offers New Hope for Hunter Syndrome
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…
Continue Reading
One-Time Gene Therapy Offers New Hope for Hunter Syndrome
UNC Researchers Report Promising Advances Toward First Brain‑Targeting Enzyme Therapy for Hunter Syndrome
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…
Continue Reading
UNC Researchers Report Promising Advances Toward First Brain‑Targeting Enzyme Therapy for Hunter Syndrome
First-in-Human Gene Therapy Offers Hope for Hunter Syndrome: Oliver’s Remarkable Turnaround
Three-year-old Oliver Chu has stunned clinicians after becoming the first person with Hunter syndrome (MPS II) to receive an experimental gene therapy and showing striking early gains. Hunter syndrome is…
Continue Reading
First-in-Human Gene Therapy Offers Hope for Hunter Syndrome: Oliver’s Remarkable Turnaround
FDA Extends Review Timeline for Denali Therapeutics’ Tividenofusp Alfa in Hunter Syndrome
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…
Continue Reading
FDA Extends Review Timeline for Denali Therapeutics’ Tividenofusp Alfa in Hunter Syndrome
source: shutterstock.com
Promising Interim Data in Phase 1/2 Hunter Syndrome Trial
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…
Continue Reading
Promising Interim Data in Phase 1/2 Hunter Syndrome Trial
source: shutterstock.com
RDLA Webinar: Updates on the VALID Act, RUSP Alignment, Newborn Screening, and Precision Medicine for Kids
On September 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
Continue Reading
RDLA Webinar: Updates on the VALID Act, RUSP Alignment, Newborn Screening, and Precision Medicine for Kids
source: pixabay.com
Discontinued Development for TAK-609, an Experimental Hunter Syndrome Therapy
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…
Continue Reading
Discontinued Development for TAK-609, an Experimental Hunter Syndrome Therapy
source: pixabay.com
FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome
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…
Continue Reading
FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome
Source: Pixabay.com
Hunterase ICV for MPS II Earns Orphan Drug Status in EU
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…
Continue Reading
Hunterase ICV for MPS II Earns Orphan Drug Status in EU
Afraid of the Doctor excerpt by Melissa J. Hogan
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…
Continue Reading
Afraid of the Doctor excerpt by Melissa J. Hogan
ANGEL AID CARES
Passion with a Purpose: How ANGEL AID CARES Uplifts Rare Mothers
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.…
Continue Reading
Passion with a Purpose: How ANGEL AID CARES Uplifts Rare Mothers
rawpixel / Pixabay
Additional Data Available on RGX-121 for MPS II
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…
Continue Reading
Additional Data Available on RGX-121 for MPS II
source: shutterstock
Rare Classroom: Hunter Syndrome
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…
Continue Reading
Rare Classroom: Hunter Syndrome
https://unsplash.com/photos/3KGF9R_0oHs
DNL310 for Hunter Syndrome Granted Fast Track Designation
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…
Continue Reading
DNL310 for Hunter Syndrome Granted Fast Track Designation
PhotoMIX-Company / Pixabay
New Data Presented on Gene Therapy for Hunter Syndrome
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…
Continue Reading
New Data Presented on Gene Therapy for Hunter Syndrome
https://pixabay.com/en/africa-zebra-black-and-white-safari-1203936/
Natural Language Processing is Changing How We Research Rare Diseases
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…
Continue Reading
Natural Language Processing is Changing How We Research Rare Diseases
