{"id":59201,"date":"2020-10-09T14:45:57","date_gmt":"2020-10-09T18:45:57","guid":{"rendered":"https:\/\/rarediseases.org\/nord-highlights-new-era-of-innovation-and-public-health-awareness-in-virtual-2020-rare-diseases-and-orphan-products-breakthrough-summit\/"},"modified":"2022-12-02T08:13:32","modified_gmt":"2022-12-02T13:13:32","slug":"nord-highlights-new-era-of-innovation-and-public-health-awareness-in-virtual-2020-rare-diseases-and-orphan-products-breakthrough-summit","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/nord-highlights-new-era-of-innovation-and-public-health-awareness-in-virtual-2020-rare-diseases-and-orphan-products-breakthrough-summit\/","title":{"rendered":"NORD Highlights New Era of Innovation and Public Health Awareness in Virtual 2020 Rare Diseases and Orphan Products Breakthrough Summit"},"content":{"rendered":"

Washington, DC, October\u00a0<\/span><\/b>9<\/span><\/b>, 20<\/span><\/b>20<\/span><\/b>\u2014On October 8 and 9, t<\/span>he National Organization for Rare Disorders (NORD<\/span>\u00ae<\/span>)\u00a0<\/span>virtually\u00a0<\/span>hosted<\/span>\u00a0the\u00a0<\/span>20<\/span>20<\/span>\u00a0<\/span>Rare Diseases and Orphan Products Breakthrough Summit<\/span><\/a>,<\/span>\u00a0<\/span>the first time the\u00a0<\/span>organization representing over 25 million Americans impacted by rare diseases<\/span>\u00a0has presented the\u00a0<\/span>confer<\/span>ence exclusively online<\/span>.<\/span>\u00a0<\/span>Nearly\u00a0<\/span>900\u00a0<\/span>registrants<\/span>\u00a0from<\/span>\u00a0<\/span>more than 20 countries around the world<\/span>\u00a0gathered\u00a0<\/span>online\u00a0<\/span>for the\u00a0<\/span>annual\u00a0<\/span>event, the\u00a0<\/span>most\u00a0<\/span>impactful<\/span>\u00a0<\/span>rare disease conference i<\/span>n the United States.<\/span>\u00a0<\/span><\/p>\n

Each year t<\/span>he NORD Summit<\/span>\u00a0<\/span>brings\u00a0<\/span>together participants\u00a0<\/span>spanning<\/span>\u00a0the rare community<\/span>,<\/span>\u00a0<\/span>including<\/span>\u00a0rare disease experts<\/span>\u00a0and leaders from<\/span>\u00a0<\/span>patient advocacy groups, government, industry and academia<\/span>\u00a0<\/span>to discuss the most current and\u00a0<\/span>critical<\/span>\u00a0topics related to rare diseases and orphan products. The 20<\/span>20<\/span>\u00a0theme<\/span>, \u201cEntering a New Era,\u201d<\/span>\u00a0<\/span>highlighted the current climate of scientific innovation that allows us to envision life-altering or even curative treatments<\/span>, as well as today\u2019s awareness of the importance of addressing global public health challenges.<\/span>\u00a0<\/span><\/p>\n

The\u00a0<\/span>two<\/span>–<\/span>day event<\/span>\u00a0kicked off\u00a0<\/span>on<\/span>\u00a0<\/span>Thursday<\/span>\u00a0morning<\/span>\u00a0<\/span>with a<\/span>\u00a0<\/span>greeting from NORD\u2019s President and CEO, Peter L. Saltonstall, followed by a<\/span>\u00a0moving<\/span>\u00a0<\/span>p<\/span>atient\/<\/span>c<\/span>aregiver\u00a0<\/span>o<\/span>pening\u00a0<\/span>a<\/span>ddress<\/span>.<\/span>\u00a0\u201c<\/span>Innovation Transforms Lives<\/span>\u201d\u00a0<\/span>featur<\/span>ed<\/span>\u00a0<\/span>Jennifer Beck of the American Porphyria\u00a0<\/span>Foundation<\/span>,\u00a0<\/span>Amy Medina of Cure SMA, and Mark Sleeper, an individual living with cystic fibrosis, discussing<\/span>\u00a0how their lives have been<\/span>\u00a0<\/span>dramatically\u00a0<\/span>altered<\/span>\u00a0by\u00a0<\/span>recent medical advancements.<\/span>\u00a0<\/span><\/p>\n

In his<\/span>\u00a0<\/span>keynote<\/span>,<\/span>\u00a0<\/span>US Food and Drug Administration (<\/span>FDA<\/span>)<\/span>\u00a0Commissioner Stephen Hahn, MD<\/span>\u00a0<\/span>touched on\u00a0<\/span>topics\u00a0<\/span>including<\/span>\u00a0COVID-19,\u00a0<\/span>the collaboration between\u00a0<\/span>NORD and FDA<\/span>\u00a0on patient\u00a0<\/span>l<\/span>istening\u00a0<\/span>s<\/span>essions<\/span>\u00a0and in\u00a0<\/span>rare disease\u00a0<\/span>research<\/span>,\u00a0<\/span>and<\/span>\u00a0<\/span>the 51 orphan indications covering a broad range of<\/span>\u00a0rare diseases<\/span>\u00a0<\/span>that have been\u00a0<\/span>approved by FDA through July of this year.<\/span>\u00a0<\/span>\u201c<\/span>It is essential that even as we continue to support the development of safe and effective treatments and vaccines for COVID-19 during this\u00a0<\/span>public health<\/span>\u00a0emergency<\/span>,<\/span>\u00a0we also advance FDA\u2019s other priorities including the\u00a0<\/span>development<\/span>\u00a0of treatments for patients w<\/span>ith rare diseases<\/span>,<\/span>\u201d<\/span>\u00a0said Dr. Hahn<\/span>, who went on to praise NORD for\u00a0<\/span>the organization\u2019s\u00a0<\/span>\u201c<\/span>important role in s<\/span>hining a light on, encouraging and forging opportunities to advance new and groundbreaking scientific research in areas where there might<\/span>\u00a0otherwise not be attention and action.\u201d<\/span>\u00a0<\/span>\u00a0<\/span><\/p>\n

Other highlights\u00a0<\/span>from day one of the\u00a0<\/span>Summit<\/span>\u00a0<\/span>included\u00a0<\/span>discussions<\/span>\u00a0on what has been learned from COVID-19 and how it can be applied for a stronger health care system,\u00a0<\/span>perspectives from Capitol Hill staffers on\u00a0<\/span>2020<\/span>\u00a0and what is ahead<\/span>,\u00a0<\/span>and\u00a0<\/span>breakout<\/span>\u00a0sessions on topics ranging from\u00a0<\/span>designing a regulatory-ready natural history study,<\/span>\u00a0virtual fundraising and grassroots advocacy\u00a0<\/span>to<\/span>\u00a0<\/span>advancing access to diagnostic testing<\/span>,<\/span>\u00a0<\/span>streamlining orphan drug development and\u00a0<\/span>the latest news on gene therapy and genome editing research.\u00a0<\/span>\u00a0<\/span><\/p>\n

Sponsors of the 2020 NORD Summit included\u00a0<\/span>Eversana<\/span>, Insmed, Retrophin and Takeda.\u00a0<\/span>T<\/span>o sign up to receive communications on the 2021\u00a0<\/span>Rare Diseases and Orphan Products Breakthrough Summit<\/span>,\u00a0<\/span>please click\u00a0<\/span>here<\/span>.<\/span>\u00a0<\/span><\/p>\n

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Tonight\u00a0<\/span>at 7:00pm EDT,\u00a0<\/span>NORD will premiere\u00a0<\/span>a\u00a0<\/span>streaming version of the Rare Impact Awards,<\/span>\u00a0with<\/span>\u00a0<\/span>2020 honorees includ<\/span>ing<\/span>\u00a0legislators, advocates,<\/span>\u00a0researchers, physicians and a rock and roll legend<\/span>, all\u00a0<\/span>being celebrated for\u00a0<\/span>making extraordinary strides for the rare disease community<\/span>.\u00a0<\/span>Janet Woodcock, MD,\u00a0<\/span>Director of the Center for Drug Evaluation and Research,\u00a0<\/span>FDA<\/span>, will be honor<\/span>ed with a Lifetime Achievement Award. The 2020 Rare Impact\u00a0<\/span>A<\/span>wards\u00a0<\/span>program is<\/span>\u00a0free\u00a0<\/span>and open to the public,\u00a0<\/span>with<\/span>\u00a0<\/span>registration<\/span><\/a>\u00a0required.<\/span>\u00a0<\/span><\/p>\n

\u00a0<\/span>#\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0 #\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0\u00a0 #<\/span>\u00a0<\/span><\/p>\n

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Washington, DC, October\u00a09, 2020\u2014On October 8 and 9, the National Organization for Rare Disorders (NORD\u00ae)\u00a0virtually\u00a0hosted\u00a0the\u00a02020\u00a0Rare Diseases and Orphan Products Breakthrough Summit,\u00a0the first time the\u00a0organization representing over 25 million Americans impacted … <\/p>\n

Continue reading “NORD Highlights New Era of Innovation and Public Health Awareness in Virtual 2020 Rare Diseases and Orphan Products Breakthrough Summit”<\/span><\/a><\/p>\n","protected":false},"author":1,"featured_media":13015,"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":[232,503,190,210,191,192,504,193],"tags":[989,357,683,2469,2095,2516,2096,1762,200,243,643,930,2097,2089,2032],"class_list":["post-59201","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-advocacy","category-events","category-featured-news","category-industry","category-medical","category-patients-members","category-press-releases","category-research","tag-american-porphyria-foundation","tag-capitol-hill","tag-cure-sma","tag-cystic-fibrosis-foundation","tag-eversana","tag-fda-commissioner-stephen-hahn","tag-insmed","tag-md","tag-nord-summit","tag-peter-l-saltonstall","tag-rare-diseases-and-orphan-products-breakthrough-summit","tag-rare-impact-awards","tag-retrophin","tag-takeda","tag-us-food-and-drug-administration"],"acf":[],"_links":{"self":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59201","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=59201"}],"version-history":[{"count":0,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59201\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media\/13015"}],"wp:attachment":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media?parent=59201"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/categories?post=59201"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/tags?post=59201"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}