MDA Celebrates the Approval of AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy

Celebrating the Approval of AGAMREE® (vamorolone) for Treatment of DMD [Muscular Dystrophy Association]( [Breaking News in DMD Disease Treatment]( MDA Celebrates the Approval of AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of AGAMREE (vamorolone), a structurally unique steroidal anti-inflammatory drug to treat children and adolescents living with Duchenne muscular dystrophy (DMD). AGAMREE (vamorolone) will be made available in the United States by [Catalyst Pharmaceuticals](. "Corticosteroids have been a first line treatment for DMD for many years but their utility has always been limited by the side effect profile, which includes weight gain, short stature, and decreased bone density among others. The approval of AGAMREE (vamorolone) provides people living with Duchenne, and their families, a powerful tool to treat the disease, while limiting some negative side effects associated with corticosteroids,” said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA. "MDA is extremely proud to have been involved with AGAMREE (vamorolone) since its inception. It was one of the earliest investments to be made by our venture philanthropy arm and we are gratified to see that paying off in the form of a viable new therapy for Duchenne patients and the community.” [Read the Full Announcement]( “Muscular Dystrophy Association has been invested in this treatment since the beginning and they truly made the program possible ,” said Eric Hoffman, Ph.D., Associate Dean, Research and Research Development; Professor, Pharmaceutical Services at Binghamton University. “The main concern was getting an effective treatment to children as quickly as possible. This was truly a community effort and getting a multitude perspectives and feedback from MDA and others helped make this a reality for so many families.” [Learn More]( Your Support Makes This Possible For Neekeesha and Jay Griffin of Harrisburg, North Carolina, the approval marks the culmination of over six years of experience with vamorolone. Their now 14-year-old son Franklin was the first boy enrolled in the US clinical trial for the treatment back in 2016, and he has remained on the therapy after the trial through an expanded access program. AGAMREE (vamorolone) has already had significant impacts on families, including yielding positive results for the Griffin family that receive multidisciplinary care at the MDA Care Center at Duke University. “My son was diagnosed with Duchenne muscular dystrophy, and he was the first person dosed with AGAMREE (vamorolone) right before his seventh birthday. Time wasn’t our best friend, and we were willing to try this treatment because of the care of Dr. Smith [Edward Smith, M.D.] and the support MDA showed us,” said Jay Griffin, MDA family member. “This approval means everything to all the kids that wouldn’t otherwise have alternative options. Our son hasn’t seen any of the typical side effects that come with other steroids so we’re sticking with it.” Because of the support of our community, we’re able to continue our investment in research to make treatments like AGAMREE (vamorolone) a reality. Please consider making a gift today to help make this progress possible for years to come. [Continue the Impact]( --------------------------------------------------------------- [Facebook]( [Twitter]( [Instagram]( [LinkedIn]( [YouTube]( [MDA.org]( © 2023 Muscular Dystrophy Association, Inc.. All rights reserved. 1016 W Jackson Blvd #1073 Chicago, IL 60607 1-800-572-1717 [Unsubscribe]( | [State Fundraising Notices]( The Muscular Dystrophy Association (MDA) is a qualified 501(c)(3) tax-exempt organization. [View Online]( | [Subscription Center]( | [Privacy Policy](