{"id":282541,"date":"2025-12-16T13:39:56","date_gmt":"2025-12-16T18:39:56","guid":{"rendered":"https:\/\/rarediseases.org\/?p=282541"},"modified":"2025-12-16T14:35:20","modified_gmt":"2025-12-16T19:35:20","slug":"nord-ceo-statement-on-the-addition-of-metachromatic-leukodystrophy-mld-and-duchenne-muscular-dystrophy-dmd-to-the-recommended-uniform-screening-panel","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/nord-ceo-statement-on-the-addition-of-metachromatic-leukodystrophy-mld-and-duchenne-muscular-dystrophy-dmd-to-the-recommended-uniform-screening-panel\/","title":{"rendered":"NORD CEO Statement on the Addition of Metachromatic Leukodystrophy (MLD) and Duchenne Muscular Dystrophy (DMD) to the Recommended Uniform Screening Panel"},"content":{"rendered":"

The National Organization for Rare Disorders (NORD) applauds the Department of Health and Human Services (HHS) for adding metachromatic leukodystrophy (MLD)<\/a> and Duchenne muscular dystrophy (DMD)<\/a> to the Recommended Uniform Screening Panel (RUSP). MLD and DMD are both serious rare conditions that have taken the lives of too many children and young adults.\u00a0<\/span>\u00a0<\/span><\/p>\n

Newborn screening is a vital public health program with a long history of successful operation in the United States. Over 14,000 newborns are found to have a serious but treatable rare disorder through newborn screening each year.1<\/sup><\/span>\u00a0Babies\u00a0affected by\u00a0these conditions\u00a0appear healthy at birth, and families are often unaware that anything is wrong\u00a0until\u00a0after\u00a0a child becomes symptomatic. Screening shortly after birth will allow for early detection and intervention, a move that will save lives and give newborns affected by\u00a0MLD and DMD\u00a0the best shot at a healthy life.\u00a0We congratulate the leukodystrophy and muscular dystrophy communities, whose tireless advocacy has driven these nominations forward\u00a0through months of uncertainty.<\/span>\u00a0<\/span><\/p>\n

While newborn screening programs\u00a0operate\u00a0at the state\/territory level, the federal government plays a critical role in supporting efficiency and transparency for our nation\u2019s newborn screening system. States\u00a0ultimately determine\u00a0which conditions are screened as part of their respective newborn screening programs, but inclusion on the RUSP is\u00a0an important step\u00a0in ensuring newborns are universally screened for a particular condition. NORD is grateful for Secretary Kennedy\u2019s decision to add MLD and DMD to the recommended panel, a decision that marks\u00a0an important step\u00a0toward universal screening for these two conditions across the country.\u00a0<\/span>\u00a0<\/span><\/p>\n

It\u00a0is critical that the important work of evaluating nominations to the RUSP, reviewing evidence, and making recommendations to the Secretary does not end with these two conditions.<\/span>\u00a0<\/span><\/p>\n

We are hopeful that the Administration will continue to hear the voice of the rare community, and we\u00a0remain\u00a0steadfast in our commitment to work with policymakers on both sides of the aisle to\u00a0determine\u00a0a path forward for this lifesaving work. As new treatments and improved\u00a0screening methods become available, we must ensure that\u00a0<\/span>all newborns born in the United States can\u00a0benefit\u00a0from early detection and treatment<\/span>.\u00a0<\/span>\u00a0<\/span><\/p>\n

To build\u00a0upon this momentum, NORD\u00a0calls on\u00a0Congress to build off this important milestone to further strengthen the nation’s newborn screening system by\u00a0passing\u00a0the\u00a0Newborn Screening Saves Lives\u00a0Reauthorization\u00a0Act.\u00a0<\/span>\u00a0<\/span><\/p>\n

This bipartisan legislation would strengthen and modernize the nation\u2019s newborn screening system, provide long-term stability for the RUSP, and ensure that states, clinicians, and families have clear,\u00a0consistent guidance. By codifying these efforts, Congress can help ensure that progress made today translates into durable, nationwide impact,\u00a0so that every newborn, regardless of where they are born, has the same opportunity for early diagnosis and\u00a0timely\u00a0care.<\/span> Advocates are encouraged to contact their lawmakers in support of this bill using NORD’s action alert here<\/a>.<\/strong><\/p>\n

Pamela Gavin<\/span>
\nChief Executive Officer<\/span>
\nNational Organization for Rare Disorders (NORD)<\/span>\u00a0<\/span><\/p>\n

\"NORD
NORD CEO Pamela Gavin joined MLD families to advocate on Capitol Hill<\/figcaption><\/figure>\n

[1]\u00a0Gaviglio A, McKasson S, Singh S, Ojodu J. Infants with Congenital Diseases Identified through Newborn Screening-United States, 2018-2020. Int J Neonatal Screen. 2023;9(2):23. Published 2023 Apr 13. doi:10.3390\/ijns9020023<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"

The National Organization for Rare Disorders (NORD) applauds the Department of Health and Human Services (HHS) for adding metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD) to the Recommended Uniform … <\/p>\n

Continue reading “NORD CEO Statement on the Addition of Metachromatic Leukodystrophy (MLD) and Duchenne Muscular Dystrophy (DMD) to the Recommended Uniform Screening Panel”<\/span><\/a><\/p>\n","protected":false},"author":13,"featured_media":282542,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_relevanssi_hide_post":"","_relevanssi_hide_content":"","_relevanssi_pin_for_all":"","_relevanssi_pin_keywords":"","_relevanssi_unpin_keywords":"","_relevanssi_related_keywords":"","_relevanssi_related_include_ids":"","_relevanssi_related_exclude_ids":"","_relevanssi_related_no_append":"","_relevanssi_related_not_related":"","_relevanssi_related_posts":"","_relevanssi_noindex_reason":"","_price":"","_stock":"","_tribe_ticket_header":"","_tribe_default_ticket_provider":"","_tribe_ticket_capacity":"","_ticket_start_date":"","_ticket_end_date":"","_tribe_ticket_show_description":"","_tribe_ticket_show_not_going":false,"_tribe_ticket_use_global_stock":"","_tribe_ticket_global_stock_level":"","_global_stock_mode":"","_global_stock_cap":"","_tribe_rsvp_for_event":"","_tribe_ticket_going_count":"","_tribe_ticket_not_going_count":"","_tribe_tickets_list":"[]","_tribe_ticket_has_attendee_info_fields":false,"footnotes":""},"categories":[232,190,2428],"tags":[],"class_list":["post-282541","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-advocacy","category-featured-news","category-newborn-screening"],"acf":[],"_links":{"self":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/282541","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/users\/13"}],"replies":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/comments?post=282541"}],"version-history":[{"count":0,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/282541\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media\/282542"}],"wp:attachment":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media?parent=282541"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/categories?post=282541"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/tags?post=282541"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}