--- description: Find out why Sarepta Therapeutics Elevidys is one of the best inventions of 2024 title: Sarepta Therapeutics Elevidys: the 200 Best Inventions of 2024 image: https://static.time.com/v3/assets/bltea6093859af6183b/blt945f1eb8ebcbb050/698a8a389862f5676589ac97/elevidys-best-inventions.jpeg?branch=production&width=3840&quality=75&auto=webp&crop=16:9 --- Oct 30, 2024 # Hope for a Genetic Disease by [Don Steinberg](/author/don-steinberg-3/) ## Don Steinberg Update your preferences in [Account Settings](/account/preferences/) Close  In June, the FDA expanded its approval of Sarepta Therapeutics’ Elevidys gene therapy for Duchenne muscular dystrophy to all patients ages four and older. The one-time treatment (which costs $3.2 million) bypasses a genetic mutation to slow progression of the rare but devastating disease, an inherited condition in which children, most often boys, progressively lose muscle strength, affecting everything from the ability to walk to eventually breathing on their own. After a narrow, conditional approval for boys age 4 and 5 in 2023, the expanded approval came as a [surprise](https://www.wsj.com/tech/biotech/a-3-million-gene-therapy-maker-at-a-bargain-price-1ce508bd "undefined") since the treatment’s Phase 3 trial didn’t hit its primary [goal](https://www.cnn.com/2023/10/31/health/sarepta-duchenne-gene-therapy-trial/index.html "undefined") of statistically significant improvement on an assessment of how kids move. But the kids did do better on that metric and other secondary measurements showed statistically significant progress, and families and doctors seeking help for the often fatal condition met it with optimism. “I think the approval brings a lot of hope for Duchenne specifically, but also the platform of gene therapy for neuromuscular disease and beyond,” says Sarepta chief scientific officer Louise Rodino-Klapac, who co-invented the therapy. “It's really a pathfinder for many other gene therapies to come.” These are independent reviews of the products mentioned, but TIME receives a commission when purchases are made through affiliate links at no additional cost to the purchaser. ```json [{"@context":"https://schema.org","@type":"Article","@id":"https://time.com/collections/best-inventions-2024/7094722/sarepta-therapeutics-elevidys/","mainEntityOfPage":{"@type":"WebPage","@id":"https://time.com/collections/best-inventions-2024/7094722/sarepta-therapeutics-elevidys/"},"headline":"Sarepta Therapeutics Elevidys: the 200 Best Inventions of 2024","datePublished":"2024-10-30T11:23:00.000Z","dateModified":"2026-04-13T11:54:29.937Z","description":"Find out why Sarepta Therapeutics Elevidys is one of the best inventions of 2024","url":"https://time.com/collections/best-inventions-2024/7094722/sarepta-therapeutics-elevidys/","keywords":["Tech","E-Commerce","list","franchise","Magazine","best inventions"],"thumbnailUrl":"https://static.time.com/v3/assets/bltea6093859af6183b/blt945f1eb8ebcbb050/698a8a389862f5676589ac97/elevidys-best-inventions.jpeg?branch=production&width=1200&quality=75&auto=webp&crop=1200:675&height=675","author":[{"@type":"Person","name":"Don Steinberg","jobTitle":null,"url":"https://time.com/author/don-steinberg-3/"}],"creator":"Don Steinberg","articleSection":"Business","image":[{"@type":"ImageObject","url":"https://static.time.com/v3/assets/bltea6093859af6183b/blt945f1eb8ebcbb050/698a8a389862f5676589ac97/elevidys-best-inventions.jpeg?branch=production&width=1200&quality=75&auto=webp&crop=1200:675&height=675","width":1200,"height":675}],"publisher":{"@type":"Organization","name":"Time","url":"https://time.com/","logo":{"@type":"ImageObject","url":"https://time.com/images/logo.png","width":528,"height":156},"foundingDate":"March 3, 1923","sameAs":["https://www.facebook.com/time","https://www.instagram.com/time/?hl=en","https://twitter.com/time","https://www.pinterest.com/timemagazine"]}},{"@context":"https://schema.org","@type":"BreadcrumbList","itemListElement":[{"@type":"ListItem","position":1,"item":{"@id":"/section/business/","name":"Business"}},{"@type":"ListItem","position":2,"item":{"@id":"/tag/time-section-tech/","name":"Tech"}},{"@type":"ListItem","position":3,"item":{"@id":"https://time.com/collections/best-inventions-2024/7094722/sarepta-therapeutics-elevidys/","name":"Sarepta Therapeutics Elevidys: the 200 Best Inventions of 2024"}}]}] ```