Duchenne muscular dystrophy (DMD)

Monroe Carell Jr. Children’s Hospital at Vanderbilt begins treating patients with novel gene therapy for Duchenne muscular dystrophy

Monroe Carell Jr. Children’s Hospital at Vanderbilt’s Jonathan Soslow, MD, and Bryan Burnette, MD, recently began treating patients with a novel gene therapy for Duchenne muscular dystrophy.

TEE tool at Vanderbilt used to monitor patients before and after surgery

Risk score for muscular dystrophy

An imaging-based risk score can identify patients with Duchenne muscular dystrophy who are at greater risk of early mortality, allowing more intensive treatment and potentially prolonging life.

Javion Johnson, a patient at Children’s Hospital’s Duchenne Muscular Dystrophy Clinic, holds a plaque designating the clinic as a Certified Care Center. Others on hand include from left, his father Ronnie Johnson, mother, Xavier Verge, brother, J’zarion Williams, Andrew Sokolow, MD, Bryan Burnette, MD, Jonathan Soslow, MD, Kali Barbalena and Haleigh Southern, RN.

Vanderbilt’s Duchenne Clinic first in Tennessee to be certified

The Duchenne Muscular Dystrophy Clinic at Monroe Carell Jr. Children’s Hospital at Vanderbilt has been recognized as a Certified Duchenne Care Center Program by the Parent Project Muscular Dystrophy, and is the only center in Tennessee to receive the distinction.

Cardiac dysfunction in DMD

The protein MMP7 is elevated in blood from patients with Duchenne muscular dystrophy who have cardiac dysfunction, suggesting that it may be a biomarker for heart disease severity.

Muscular dystrophy clue

Vanderbilt investigators have discovered a role for immune system T cells in slowing the decline in skeletal muscle function in patients with Duchenne muscular dystrophy.

Personal touch: Family’s story drives home impact of research

For 30 minutes Tuesday, Terry Marlin, father of two young boys with Duchenne muscular dystrophy, was the professor, telling his story to a group of Vanderbilt University graduate students and postdoctoral fellows.