Breaking News: New FDA Approval for Duchenne Muscular Dystrophy

Celebrating the Approval of Duvyzat (givinostat) for Treatment of DMD [Muscular Dystrophy Association]( [Breaking News in DMD Disease Treatment]( Dear {NAME}, We are excited to announce a significant breakthrough in the treatment of Duchenne muscular dystrophy (DMD). Today, the [U.S. Food and Drug Administration (FDA) has approved Duvyzat (givinostat)]( to treat children and adolescents with DMD. Pier Lorenzo Puri’s group based at Sanford Burnham Prebys Medical Discovery Institute in San Diego performed the studies that led to the discovery of HDAC inhibitors (HDACi) for treatment of DMD since 2004. These studies were performed in close collaboration with the lab Puri led in Rome in Scientific Park of Castel Romano (2002-2007) and at Fondazione Santa Lucia (2008-2018). “Muscular Dystrophy Association’s funding was fundamental for the discovery of HDAC inhibitors (HDACi) in the treatment of Duchenne muscular dystrophy. We are grateful to MDA to have enabled studies that support the important pre-clinical evidence that HDACi exerted beneficial effects in the mouse model of DMD. These studies provided the initial experimental evidence that led me to contact Italfarmaco to test givinostat, as a HDACi that was already in clinical trial for other diseases and could have been easily repurposed for DMD,” said Pier Lorenzo Puri, M.D., Sanford Burnham Prebys Medical Discovery Institute. Duvyzat (givinostat) works by changing gene expression in cells, altering the three-dimensional folding of DNA. Duvyzat (givinostat) has been shown to slow the progression of DMD by reducing the decline of muscle function in people living with the disease. ITF Therapeutics will make Duvyzat (givinostat) available in the United States, signaling the start of a promising new chapter in DMD treatment. "The approval of Duvyzat (givinostat) provides another significant treatment option for people living with Duchenne muscular dystrophy,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA. “As more drugs are approved for DMD we look forward to understanding if and how they might be used in combination.” [Read the Full Announcement]( MDA’s Legacy in DMD Research Since our inception, MDA has invested over $229 million in research for Duchenne and Becker muscular dystrophy. The discovery of the dystrophin gene in the 1980s by MDA-funded research marked the beginning of our understanding of Duchenne and Becker muscular dystrophy. This discovery has been foundational in the development of treatments like Duvyzat (givinostat). Today’s celebration of this groundbreaking treatment is only possible because of the dedication and generosity of our community of supporters. Together, we are turning research into reality for people living with neuromuscular diseases. [Give Now to Continue the Momentum]( --------------------------------------------------------------- [Facebook]( [Twitter]( [Instagram]( [LinkedIn]( [YouTube]( [MDA.org]( © 2024 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](