{"id":58660,"date":"2015-11-20T20:23:33","date_gmt":"2015-11-21T01:23:33","guid":{"rendered":"https:\/\/rarediseases.org\/national-spotlight-on-fda-and-duchenne-muscular-dystrophy-what-this-means-for-everyone-with-a-rare-disease\/"},"modified":"2022-12-01T11:56:56","modified_gmt":"2022-12-01T16:56:56","slug":"national-spotlight-on-fda-and-duchenne-muscular-dystrophy-what-this-means-for-everyone-with-a-rare-disease","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/national-spotlight-on-fda-and-duchenne-muscular-dystrophy-what-this-means-for-everyone-with-a-rare-disease\/","title":{"rendered":"National Spotlight on FDA and Duchenne Muscular Dystrophy:\u00a0What This Means for Everyone with a Rare Disease"},"content":{"rendered":"

\"fda\"<\/a><\/strong>Next week, a\u00a0<\/strong>Food and Drug Administration panel of experts will hold the first of two meetings to review two drugs designed to slow the muscle wasting effects associated with Duchenne Muscular Dystrophy (DMD).\u00a0 These much-anticipated events provide hope for children and families living with this devastating and fatal disease, which currently has no treatment or cure.\u00a0\u00a0 There are also takeaways here for the wider rare community affected by the 7,000 known rare diseases\/disorders, the majority of which are without any sort of treatment.<\/p>\n

Earlier this week, STAT reporter Ed Silverman\u00a0looked<\/a>\u00a0at how the DMD community, FDA and drug developers arrived at this point.\u00a0 Pat Furlong, a NORD board member and chief executive of Parent Project Muscular Dystrophy (PPMD), a NORD member organization, cited as one reason 2012 legislation (the FDA Safety and Innovation Act) <\/em>that \u201copened the door for patient involvement.\u201d<\/p>\n

NORD advocates for, and works to facilitate, stronger patient participation in every step of the drug development process.\u00a0 This was a reoccurring topic at our recent Rare Diseases and Orphan Products Breakthrough Summit, where we covered how increased patient involvement in research via natural history studies and\u00a0registries<\/a>, clinical trial design, and working with\u00a0regulators<\/a>, along with scientific advances (see Precision Medicine), have helped to accelerate orphan drug development.\u00a0 In 2014, the FDA approved more orphan drugs than ever before.\u00a0 We are on track to exceed that number in 2015.<\/p>\n

To learn more about how you can participate in next week\u2019s events related to DMD, please visit our member organization\u2019s website:\u00a0https:\/\/community.parentprojectmd.org\/profiles\/blogs\/ad-comms-update-how-to-participate<\/a>.<\/p>\n

We will be watching\u00a0Tuesday\u00a0and believe that growing patient participation in the drug development process is one more reason for our community to be thankful this year.<\/p>\n","protected":false},"excerpt":{"rendered":"

Next week, a\u00a0Food and Drug Administration panel of experts will hold the first of two meetings to review two drugs designed to slow the muscle wasting effects associated with Duchenne … <\/p>\n

Continue reading \u00abNational Spotlight on FDA and Duchenne Muscular Dystrophy:\u00a0What This Means for Everyone with a Rare Disease\u00bb<\/span><\/a><\/p>\n","protected":false},"author":47,"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":[232,210],"tags":[286,859,244,858,857,856,643,861,860],"class_list":["post-58660","post","type-post","status-publish","format-standard","hentry","category-advocacy","category-industry","tag-duchenne-muscular-dystrophy","tag-ed-silverman","tag-fda","tag-fda-safety-and-innovation-act","tag-parent-project-muscular-dystrophyk-precision-medicine","tag-pat-furlong","tag-rare-diseases-and-orphan-products-breakthrough-summit","tag-registries","tag-stat"],"acf":[],"_links":{"self":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/58660","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\/47"}],"replies":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/comments?post=58660"}],"version-history":[{"count":0,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/58660\/revisions"}],"wp:attachment":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media?parent=58660"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/categories?post=58660"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/tags?post=58660"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}