Newborn Screening for MLD
The announcement by NICE that Libmeldy© (Gene Therapy) was approved for use in the UK meant that instead of MLD Support Association UK only being able to support sufferers of MLD and their families, we were now able to move forward to promote early intervention and campaign for Newborn Screening for MLD.
This means that our message has changed from looking for a treatment for MLD, to campaigning for early diagnosis so that Gene Therapy can be accessed by newly-diagnosed sufferers. Early diagnosis is, of course, best as close to birth as possible which is why Newborn Screening is so critical. Please see some of our campaign memes below.
In December 2021 a formal application to add Metachromatic Leukodystrophy to the UK Newborn Blood Spot (NBS) programme was made to the National Screening Committee (NSC). This was an important milestone for the MLD community and one which could not have been possible without the development of both a transformational therapy for pre-symptomatic MLD patients, Libmeldy©, and a breakthrough assay for detecting MLD at birth, developed by Prof. Michael Gelb in the US. We are ever grateful for the important work and collaboration of many scientists and clinicians from around the world who have shown heroic dedication to helping MLD affected patients.