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Vainieri Huttle & Sumter Bill to Help Improve Quality of Life for Children with Hunter & Hurler Syndromes Advances in Assembly

“Let Them Be Little Act” Would Require all Newborns to Be Screened for Rare Genetic Disorder

Legislation sponsored by Assemblywomen Valerie Vainieri Huttle and Shavonda Sumter to help improve the quality of life and longevity for children suffering from rare genetic disorders known as Hunter and Hurler syndromes was advanced by an Assembly panel on Monday.

The bill (A-1101), designated as the “Let Them Be Little Act,” requires infants born in New Jersey to be screened for the lysosomal disorders known as Hunter syndrome (MPS II) and Hurler syndrome (MPS I) under the same conditions that are required for other lysosomal disorders.

“Children with these syndromes are often unable to enjoy the simple pleasures of youth because so much of their lives are occupied with undergoing medical treatments and tests rather than just being kids,” said Vainieri Huttle (D-Bergen). “The ‘Let Them Be Little Act’ would help these children do just that and hopefully increase their quality of life.”

“Early detection has vastly improved the quality of life for children suffering from many different diseases,” said Sumter (D-Bergen/Passaic). “The same can be done for children with Hunter or Hurler syndrome if we take the steps now to make screening for this genetic disorder routine for every newborn born in New Jersey.”

Hunter syndrome causes permanent, progressive damage affecting appearance, mental development, organ function, and physical abilities. Hunter syndrome occurs mainly among males when an enzyme needed to break down certain complex molecules is missing or malfunctioning. There are two subtypes: early- and late- onset Hunter syndrome.

Early-onset Hunter syndrome is more common and severe, and appears usually between two and four years of age. By late childhood, a child may suffer from a severe mental disability and may not live beyond the teenage years. Late-onset Hunter syndrome is milder, and is typically diagnosed after 10 years of age but may not be detected until adulthood. A person with late-onset Hunter syndrome may live into their 50’s.

Early screening and diagnosis of Hunter syndrome and appropriate management, through administration of prescription drugs such as ELAPRASE, would enable a child with Hunter Syndrome to live a longer and higher quality of life.

Hurler syndrome is a rare, inherited disease of metabolism in which a person cannot break down long chains of sugar molecules called glycosaminoglycans. Without the enzyme, glycosaminoglycans build up and damage organs, including the heart. Symptoms can range from mild to severe and typically begin to appear in children between the ages of 3 and 8.

Current law requires newborn screening of other lysosomal disorders, such as Krabbe, Pompe, Gaucher, Fabry, and Niemann-Pick diseases, within six months of the availability of the necessary testing methodology and the acquisition by the Department of Health of the equipment necessary to implement the expanded screening tests.

Under the bill, all infants would be screened for Hunter and Hurler syndromes beginning six months following the occurrence of all of the following:

1) the development of a reliable test or series of tests for screening newborns for MPS I & II using dried blood spots and quality assurance testing methodology for Hunter syndrome testing;

2) the availability of quality assurance materials for the MPS I & II test from the federal Centers for Disease Control and Prevention;

3) the inclusion of newborn screening for MPS I & II in the Recommended Uniform Screening Panel of the United States Secretary of Health and Human Services’ Advisory Committee on Heritable Disorders in Newborns and Children, after the committee’s evidence review of newborn screening for MPS I & II;

4) the review by the Department of Health of the proposed test; and

5) the acquisition of equipment necessary to implement the expanded screening.

The bill was approved by the Assembly Women and Children Committee and now awaits consideration by the full Assembly.