The Muscular Dystrophy Association launched a new event series in 2017, MDA Discovery & Impact Events, to bring greater awareness to and educate families and supporters about the progress being made in the neuromuscular disease space.

Industry and health care leaders, as well as families benefitting from research advances, served as guest speakers, highlighting the safe and effective treatment options now available to MDA families, including three new drugs that can trace their origins directly to MDA research dollars. In 2016, the FDA approved Exondys 51 for some forms of Duchenne muscular dystrophy (DMD) and Spinraza for spinal muscular atrophy (SMA) — the first drugs approved to treat these diseases. In 2017, Emflaza was also approved to treat DMD.

The community-focused events brought together key stakeholders and community leaders to celebrate progress made and learn more about the current work being done in an effort to bring forth more advances and treatments.

Thanks to the generous support received to date from Biogen, PTC Therapeutics and Santhera, MDA hosted events in the following cities: Chicago, Boston, New York, Dallas and Houston. There are plans to host more events this year, including an event in Washington, D.C. scheduled for Feb. 8, 2018 at the National Press Club.”

Washington, D.C. Discovery & Impact Event

Register today for this event scheduled for Thursday, February 8, 2018 at the National Press Club.

Boston Discovery & Impact Event

Held Oct. 17 at the UMass Club, the event’s guest speakers included:

• Dr. Basil Darras – associate neurologist-in-chief, chief of the Division of Clinical Neurology, director of the Neuromuscular Center and Spinal Muscular Atrophy Program at Boston Children’s Hospital and professor of neurology at Harvard Medical School.
• Jenn McNary – mother of four and fierce advocate in the DMD community, recipient of Ryan’s Quest Ryan’s Hero Award in 2013, a nominee for the Global Genes Champion of Hope Award in 2014 and recipient of the 2017 Meyer-Whalley Instrument of Change Award.

Dallas Discovery & Impact Event

Held Oct. 26 at the La Cima Club, the event’s guest speakers included:

• Dr. Diana Castro – assistant professor in the Department of Pediatrics, Neurology and Neurotherapeutics at the University of Texas Southwestern and director of the MDA Care Center and Children’s Health. Hear her presentation on cures and therapies for SMA and DMD.
• Dr. Susan Iannaccone – professor in the Department of Pediatrics, Neurology and Neurotherapeutics at the University of Texas Southwestern (UTSW), associate director of the UTSW Wellstone Muscular Dystrophy Center and director of the MDA Care Center at Children’s Health. Hear her presentation about research into cures and therapies for DMD.
• Sandy Spoonemore – Sandy was diagnosed with SMA at the age of 2 and has used a wheelchair her entire life. In 2016, Sandy was crowned Ms. Wheelchair Texas and tirelessly traveled the state with her platform to encourage individuals, families and companies to see that everyone has value and a purpose. Sandy works as an independent sales director with Mary Kay Cosmetics and has been honored with many awards recognizing her work ethic, innovation and marketing talents, including  being named the Entrepreneur of the Year from the Governor of Texas. Recently, Sandy completed her four loading doses of the Spinraza treatment and has seen very promising results. Hear her presentation.
• Listen to the event’s question and answer session with guest speakers.

New York City Discovery & Impact Event

Held Nov. 7 at the Cornell Club New York, the event’s guest speakers included:

• Dr. Michio Hirano – chief of the Neuromuscular Division and co-director of the MDA Care Center at Columbia University Medical Center and director of the H. Houston Merritt Center for Muscular Dystrophy and Related Diseases. Hear his presentation on  developments in genetic research into cures and treatments for muscular dystrophy.
• Lucinda Rosso – MDA advocate and mother to Adelina (Addy) who lives with central core disease. Hear her talk about the impact the disease has had on their family and how she has found support from MDA.
• Listen to the event’s question and answer session with guest speakers.

Chicago Discovery & Impact Event

Held Nov. 8 at the Chicago Symphony Orchestra, the event’s guest speakers included:

• Dr. Nancy L. Kuntz – medical director of the Mazza Foundation Neuromuscular Disorders Program, the PPMD Duchenne Care center and the MDA Care Center at Lurie Children’s Hospital and professor of pediatrics and neurology at Northwestern Feinberg School of Medicine. Hear her presentation.
• Hally Goldstein – MDA advocate and mother to Yehuda, who lives with Becker muscular dystrophy. Listen to her discussion of the impact the disease has had on her family.
• Listen to the event’s question and answer session with guest speakers.

Houston Discovery & Impact Event

Held Nov. 16 at the Health Museum, the event’s guest speakers include:

• Dr. Stanley H. Appel – chair of the Department of Neurology and co-director of the Houston Methodist Neurological Institute at Houston Methodist Hospital and professor of neurology at Weill Cornell Medical College. Hear his presentation.
• Dr. Timothy Edward Lotze – associate professor of pediatric neurology and director of the pediatric neurology residency training program at Baylor College of Medicine and director of the MDA Pediatric Care Center at Texas Children’s Hospital. Hear his presentation.
• Dyla Gutierrez – MDA advocate and mother to Dyla (little D) who is living with SMA and recently received four loading doses of Spinraza. She’s seen very promising results. Listen to her share the impact and progress occurring in her family.
• Listen to the event’s question and answer session with all guest speakers.