ConnectING FAMILIES
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PO Box 2866

Bala Cynwyd PA

19004 USA

info@curemld.com

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Support for Cure MLD comes from an unrestricted grant from Orchard Therapeutics. 

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Why is newborn screening such an important part of our work here at Cure MLD?

In the excitement of a child’s birth, most new parents hardly notice the drops of blood that are collected for newborn screening to test for dozens of serious, life-threatening disorders. Too few physicians and patients understand how important a tool it is for saving children’s lives. Except for vaccinations, no other public health initiative has been more impactful.

 

In the US, nearly four million newborns are screened at birth each year for dozens of disorders. Experts believe newborn screening saves 12,000 children’s lives in the United States annually.

The reality is that the recent breakthroughs in gene therapy are offering new hope to patients with MLD, but these treatments are most effective if children get diagnosed before the onset of symptoms in the first year of life.  

One of the most serious challenges for newborn screening is the scale. Developing a screening test that can be performed efficiently and cost-effectively on millions of babies is a massive undertaking. In the US, the Every Life Foundation for Rare Diseases has worked to eliminate these legislative delays by ensuring states screen for recommended diseases within a set time frame, but progress is depressingly slow.  

 

As of this writing, the only leukodystrophy included on the Recommended Uniform Screening Panel (RUSP) is adrenoleukodystrophy (ALD or the Lorenzo’s Oil disease). ALD newborn screening advocate Elisa Seeger frequently says in her work with elected officials, “saving a child’s life should not depend on where you live.”

The University of Washington’s Dr. Michael Gelb and the MLD Foundation's Dean and Teryn Suhr, have spent the last five years developing a newborn screening for MLD.  By 2020, Cure MLD will be working with researchers, patient advocacy leaders and industry stakeholders to pilot an MLD newborn screening internationally. This is a crucial step in saving children’s lives and ensuring that breakthroughs in gene therapy and other treatments can reach patients in time.

Learn more about the challenges and obstacles for newborn screening here

Please let us know if you would like help Cure MLD ensure that every child in the world is screened for MLD at birth!

Elisa Seeger and Aidan.JPG

I am a caregiver or an adult patient with MLD and I would like to speak to other adult patients/caregivers.

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