When Raniya Scott was just two years old, her world changed with an unexpected diagnosis: Duchenne muscular dystrophy (DMD), a group of diseases that causes muscles to weaken and break down over time. The news was overwhelming for her young mother, and it puzzled her doctors, as this rare and progressive genetic disorder primarily affects boys, not girls.<\/p>\n\n\n\n
Raniya\u2019s DMD affected everything from walking and breathing to blood circulation. For 11 years, Raniya has depended on medication, physical therapy, and a wheelchair to help manage her worsening symptoms.<\/p>\n\n\n\n
But now, a new gene therapy is offering the family hope. Raniya, now 16, is the first girl in the world to receive the groundbreaking gene therapy, thanks to physicians at UHealth \u2013 University of Miami Health System and Holtz Children\u2019s Hospital. Additionally, her younger brother, Amir Monstanto, 8 \u2013 who also has DMD \u2013 received the treatment alongside her. [Read More: University of Miami Health System and Holtz Children\u2019s Hospital Physicians Evaluated the Effectiveness of ELEVIDYS<\/a>]<\/p>\n\n\n\n \u201cI have been working in neurogenetics for 30 years, and it\u2019s amazing to see the dream of gene therapy being realized,\u201d Dr. Lopez-Alberola said. \u201cIt\u2019s a wonderful opportunity to treat these patients and see the impact it\u2019s having. There is much to celebrate.\u201d<\/p>\n\n\n\n Read More<\/a><\/p>\n\n\n\n When Raniya Scott was just two years old, her world changed with an unexpected diagnosis: Duchenne muscular dystrophy (DMD), a group of diseases that causes muscles to weaken and break down over time. The news was overwhelming for her young mother, and it puzzled her doctors, as this rare and progressive genetic disorder primarily affects […]<\/p>\n","protected":false},"author":1,"featured_media":2121,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[33,61,64,65],"class_list":["post-2117","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-elevidys","tag-gene-therapy","tag-raniya-scott","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/posts\/2117","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/comments?post=2117"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/posts\/2117\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/media\/2121"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/media?parent=2117"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/categories?post=2117"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/tags?post=2117"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}Also Read Article: Frequently Asked Questions About Elevidys<\/a><\/h3>\n","protected":false},"excerpt":{"rendered":"