{"id":59432,"date":"2022-08-01T12:00:38","date_gmt":"2022-08-01T16:00:38","guid":{"rendered":"https:\/\/rarediseases.org\/nord-to-host-historic-fda-meeting-on-galactosemia-in-partnership-with-the-galactosemia-foundation\/"},"modified":"2022-08-01T12:00:38","modified_gmt":"2022-08-01T16:00:38","slug":"nord-to-host-historic-fda-meeting-on-galactosemia-in-partnership-with-the-galactosemia-foundation","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/nord-to-host-historic-fda-meeting-on-galactosemia-in-partnership-with-the-galactosemia-foundation\/","title":{"rendered":"NORD to Host Historic FDA Meeting on Galactosemia, in Partnership with the Galactosemia Foundation"},"content":{"rendered":"

A historic, virtual meeting of the galactosemia community, FDA, and drug development leaders will be hosted on Thursday, September 1<\/span><\/i>st<\/span><\/i>\u00a0<\/span><\/i>\u00a0<\/span><\/h3>\n

Galactosemia is a rare, hereditary disorder that affects the body\u2019s ability to convert galactose to glucose. <\/span>Galactosemia<\/span><\/a> affects 1 in every 40,000 to 1 in 60,000 newborns around the world<\/span>. The <\/span>Galactosemia Foundation<\/span><\/a> is a non-profit, voluntary health organization that has made it their mission to maximize the potential for the development of individuals with galactosemia.<\/span>\u00a0<\/span><\/p>\n

To share community perspectives on this rare disease, NORD is proud to partner with The Galactosemia Foundation to organize and host an upcoming externally led Patient-Focused Drug Development (EL-PFDD) meeting. An EL-PFDD is an innovative event that provides an opportunity for families and patients to share critical information about the impact of a rare disease on their daily lives and provide valuable insight for FDA and other key stakeholders, including researchers, medical product developers and health care providers.<\/span>\u00a0<\/span><\/p>\n

The PFDD will be hosted virtually on <\/span>Thursday, September 1 from 11am-3:30pm ET<\/span><\/b>.<\/span>\u00a0<\/span><\/p>\n

Galactosemia patient and caregiver stories are powerful, and their perspectives are critical to the decisions made by drug developers. This is an extraordinary opportunity to listen to medical and patient discussion and storytelling, as well to participate in live, online polling and Q&A.\u202f Anyone affected by galactosemia, as well as researchers, clinicians and other supporters are invited to attend. <\/span>Registration is now open<\/span><\/b><\/a>!<\/span>\u00a0<\/span><\/p>\n

PFDDs are\u202fone of the most important\u202fways\u202fthe voice of rare disease patients and caregivers can be heard and utilized in decision making. NORD has applied to the FDA and has been granted approval to host an EL-PFDD meeting focused on several rare diseases, including pyruvate kinase deficiency (PKD) in 2019, Krabbe disease in 2020, Post-Transplant Lymphoproliferative Disorder (PTLD) in May 2022, and\u202fGalactosemia this September. Each year, NORD allocates resources and provides additional guidance and leadership to help a few groups\u202fand disease communities with need in\u202fhosting\u202fPFDDs and bring patient experiences directly to decisionmakers.\u202f<\/span>\u00a0<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"

A historic, virtual meeting of the galactosemia community, FDA, and drug development leaders will be hosted on Thursday, September 1st\u00a0\u00a0 Galactosemia is a rare, hereditary disorder that affects the body\u2019s … <\/p>\n

Continue reading “NORD to Host Historic FDA Meeting on Galactosemia, in Partnership with the Galactosemia Foundation”<\/span><\/a><\/p>\n","protected":false},"author":1,"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":[192],"tags":[270,2306,244,3500,1065,2767,404,2304],"class_list":["post-59432","post","type-post","status-publish","format-standard","hentry","category-patients-members","tag-drug-development","tag-el-pfdd","tag-fda","tag-galactosemia","tag-galactosemia-foundation","tag-patient","tag-patient-focused-drug-development","tag-pfdd"],"acf":[],"_links":{"self":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59432","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\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/comments?post=59432"}],"version-history":[{"count":0,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59432\/revisions"}],"wp:attachment":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media?parent=59432"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/categories?post=59432"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/tags?post=59432"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}