{"id":279397,"date":"2025-06-05T12:05:04","date_gmt":"2025-06-05T16:05:04","guid":{"rendered":"https:\/\/rarediseases.org\/?p=279397"},"modified":"2025-06-05T14:23:28","modified_gmt":"2025-06-05T18:23:28","slug":"first-rare-disease-therapy-developed-from-the-nord-iamrare-patient-registry-secures-fda-approval","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/first-rare-disease-therapy-developed-from-the-nord-iamrare-patient-registry-secures-fda-approval\/","title":{"rendered":"First Rare Disease Therapy Developed with a NORD IAMRARE Patient Registry Secures FDA Approval"},"content":{"rendered":"

QUINCY, Mass., June 5, 2025<\/strong> \u2013 The National Organization for Rare Disorders (NORD\u00ae<\/sup>) proudly announces a major milestone: for the first time, patient data collected through the NORD IAMRARE\u00ae<\/sup> patient registry platform<\/a> has played a pivotal role in securing U.S. Food and Drug Administration (FDA) approval for a groundbreaking therapy.<\/p>\n

VYKAT TM<\/sup> XR (diazoxide choline) extended-release tablets, which are manufactured by Soleno Therapeutics, Inc. (NASDAQ: SLNO), are the first approved treatment to address hyperphagia, or excessive hunger, in both adults and children diagnosed with Prader-Willi syndrome (PWS)<\/a>. PWS is a complex genetic disorder that begins with low muscle tone and feeding difficulties in infancy and progresses to insatiable hunger, obesity, and cognitive challenges throughout life.<\/p>\n

The Foundation for Prader-Willi Research<\/a>, leveraging NORD\u2019s IMARARE platform, led a pivotal study collecting essential patient data that was presented to the U.S. Food and Drug Administration (FDA) as part of the approval process for this treatment. On March 26, 2025, the FDA approved VYKATTM<\/sup> XR, for use in the United States for the treatment of hyperphagia in adults and children 4 years of age and older with PWS.<\/p>\n

With over 95% of rare diseases lacking an approved treatment, the IAMRARE platform is proving to be a powerful tool in the search for therapies and cures.<\/strong><\/p>\n

\u201cNORD is committed to driving advances for the rare community through initiatives such as the IAMRARE patient registry platform,\u201d said Pamela K. Gavin, NORD Chief Executive Officer<\/strong>. \u201cFor more than 40 years, we\u2019ve brought together patients, researchers, clinicians, and industry together to accelerate research and the development of new treatments. We are proud to celebrate this new therapy and continue supporting rare disease science and innovation.\u201d<\/p>\n

Through IAMRARE, patient organizations and researchers can launch studies to collect and analyze the patient-reported data essential for drug development. Designed with input from clinicians, researchers, and the FDA, IAMRARE enables deep patient engagement to drive innovation in rare disease research and treatments.<\/p>\n

\u201cFor years, families and researchers have worked towards a treatment option that truly addresses the complexities of PWS,\u201d said Susan Hedstrom, Executive Director of the Foundation for Prader-Willi Research, a NORD Member<\/a> patient advocacy organization.<\/strong> \u201cLeveraging the IAMRARE platform allowed the Foundation to take control of our data and closely collaborate with the researchers who developed this historic treatment. We appreciate NORD\u2019s leadership and role in supporting this incredible milestone for our community.\u201d<\/p>\n

As of Dec. 31, 2024, IAMRARE hosted 47 patient registries representing approximately 165 rare conditions with nearly 17,600 patients enrolled.<\/p>\n

To learn more about NORD\u2019s IAMRARE, visit iamrare.org<\/a>.<\/p>\n

About the National Organization for Rare Disorders (NORD)<\/strong><\/p>\n

With a 42-year legacy of advancing care, treatments and policy, NORD\u00ae<\/sup> is the leading and longest-standing patient advocacy organization for the more than 30 million Americans living with a rare disease. An independent 501(c)(3) nonprofit, NORD is dedicated to individuals with rare diseases and the organizations that serve them. NORD, alongside its 350+ patient organization members, is committed to improving health and well-being through research, advocacy, and innovation.<\/p>\n

About the Foundation for Prader-Willi Research (FPWR)<\/strong><\/p>\n

FPWR was established in 2003 by a small group of parents, with a mission to eliminate the challenges of Prader-Willi syndrome through the advancement of research and therapeutic development. Through a world-class grants program, translational research programs, and the development of PWS research tools, FPWR plays an active role in research to advance knowledge and develop treatments that will improve the health and well-being of those with PWS. To learn more about the Foundation for Prader-Willi Research, visit www.fpwr.org<\/a>.<\/p>\n","protected":false},"excerpt":{"rendered":"

QUINCY, Mass., June 5, 2025 \u2013 The National Organization for Rare Disorders (NORD\u00ae) proudly announces a major milestone: for the first time, patient data collected through the NORD IAMRARE\u00ae patient … <\/p>\n

Continue reading “First Rare Disease Therapy Developed with a NORD IAMRARE Patient Registry Secures FDA Approval”<\/span><\/a><\/p>\n","protected":false},"author":13,"featured_media":0,"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":[504,190,192,2722,193,4138],"tags":[],"class_list":["post-279397","post","type-post","status-publish","format-standard","hentry","category-press-releases","category-featured-news","category-patients-members","category-registries","category-research","category-researchregistries"],"acf":[],"_links":{"self":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/279397","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=279397"}],"version-history":[{"count":0,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/279397\/revisions"}],"wp:attachment":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media?parent=279397"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/categories?post=279397"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/tags?post=279397"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}