---
title: Rare Disease Treatment Unexpectedly Rejected by the FDA
description: Rare Disease Treatment Unexpectedly Rejected by the FDA
---

[News ](https://www.yourway.com/news)

# [Rare Disease Treatment Unexpectedly Rejected by the FDA](https://www.yourway.com/news/rare-disease-treatment-unexpectedly-rejected-by-the-fda)

 Written by [Yourway](https://www.yourway.com/news/author/yourway) | September 4, 2019

In a surprise [decision](https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-receives-complete-response-letter-us-food), the U.S. Food and Drug Administration has rejected Sarepta’s experimental exon-skipping drug Vyondys 53 (golodirsen) for the treatment of Duchenne muscular dystrophy, citing risks of both infection and kidney toxicity. Yourway is dedicated to supporting clinical trials for advanced therapies for orphan diseases.

[View full post](https://www.yourway.com/news/rare-disease-treatment-unexpectedly-rejected-by-the-fda)

```json
{
  "@context" : "http://schema.org",
  "@type" : "BlogPosting",
  "author" : {
    "@type" : "Person",
    "name" : "Yourway"
  },
  "dateModified" : "2019-09-03T20:37:42.713Z",
  "datePublished" : "2019-09-04T12:00:00Z",
  "headline" : "Rare Disease Treatment Unexpectedly Rejected by the FDA",
  "image" : {
    "@type" : "ImageObject",
    "height" : 960,
    "url" : "https://cdn2.hubspot.net/hubfs/5402331/News%20and%20Articles%20Banners/iStock-1169753157.jpg",
    "width" : 1440
  },
  "mainEntityOfPage" : "https://www.yourway.com/news/rare-disease-treatment-unexpectedly-rejected-by-the-fda",
  "publisher" : {
    "@type" : "Organization",
    "logo" : {
      "@type" : "ImageObject",
      "height" : 60,
      "url" : "/hs/hsstatic/content_shared_assets/static-1.4092/img/default-amp-logo.png",
      "width" : 60
    },
    "name" : "News"
  }
}
```