The North American Pediatric Renal Trials and Collaborative Studies and Horizon Pharma plc Announce New Long-Term Cystinosis Registry
The North American Pediatric Renal Trials and Collaborative Studies and Horizon Pharma plc Announce New Long-Term Cystinosis Registry
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“Initial cystinosis natural history studies were conducted decades ago
by the
People living with cystinosis under the age of 25, with consent from their guardian, can be enrolled into the registry by their physician. All of the patient data will be blinded, which means that individual names or any other information identifying a specific person will not be made available to those exploring registry data. Any person under the age of 25 can be enrolled in the study, regardless of what therapies or medications they are receiving.
“This registry will help us better understand cystinosis, identify
management improvements, and explore new treatment options that arise,”
said
While Horizon provided NAPRTCS financial support to develop the registry, the company does not own any of the data – it belongs entirely to the medical community and is accessible through a securely protected online portal managed by NAPRTCS. Interested healthcare professionals should visit www.naprtcs.org.
“This registry is an example of a prioritized initiative based on input
we’ve heard from physicians and researchers,” said
About Cystinosis
Nephropathic cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body. If untreated, elevated cystine accumulation leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death. It is estimated that only about 2,000 people worldwide are currently diagnosed with nephropathic cystinosis. Nephropathic or “classic infantile” cystinosis – the most common and most severe form of the disease – is typically diagnosed in infancy and requires lifelong cystine depleting therapy.1
About NAPRTCS
The North American Pediatric Renal Trials and Collaborative Studies
(NAPRTCS) is a research effort organized in 1987. The NAPRTCS patient
registry follows the clinical course and natural history of children
with renal dysfunction from participating sites across
Horizon Pharma plc is focused on researching, developing and
commercializing innovative medicines that address unmet treatment needs
for rare and rheumatic diseases. By fostering a growing pipeline of
medicines in development and exploring all potential uses for currently
marketed medicines, we strive to make a powerful difference for
patients, their caregivers and physicians. For us, it’s personal: by
living up to our own potential, we are helping others live up to theirs.
For more information, please visit www.horizonpharma.com,
follow us @HZNPplc
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Forward-Looking Statements
This press release contains forward-looking statements, including
statements regarding the potential benefits of the cystinosis registry.
Forward-looking statements speak only as of the date of this press
release and
1. https://ghr.nlm.nih.gov/condition/cystinosis.
Accessed
C-HZN-00107
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Source:
Horizon Pharma plc
U.S. Media Contact:
Matt Flesch
Executive
Director, Product Communications
media@horizonpharma.com