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Pamrevlumab, a Fully Human Monoclonal Antibody Targeting Connective Tissue Growth Factor, for Non-Ambulatory Patients with Duchenne Muscular Dystrophy
This Phase II, single-arm trial assessed intravenous infusions of pamrevlumab in 15 non-ambulatory patients for 2 years. The treatment is a fully human monoclonal antibody binding to connective tissue growth factor (CTGF). Results from this study found the rate of muscle function decline was slower in treated versus non-treated patients. This anti-GTGF therapy represents a potential treatment for DMD.
Multi-Laboratory Evaluation of Prototype Dried Blood Spot Quality Control Materials for Creatine Kinase-MM Newborn Screening Assays
The Newborn Screening Quality Assurance Program (NSQAP) at the U.S. Centers for Disease Control and Prevention (CDC) produced a set of seven prototype dried blood spot (DBS) reference materials spiked with varying levels of creatine kinase MM isoform (CK-MM). These DBS were evaluated over a 3-week period by CDC, NYS, and RTI, all using the same CK-MM isoformspecific fluoroimmunoassay.
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial
This international, randomized 2 : 1, placebo-controlled, phase 3 study in patients ≥ 4–<8 years old with DMD due to any dystrophin mutation examined the effect of edasalonexent (100 mg/kg/day) compared to placebo over 52weeks.
MDA 2022 Conference Presentation: Phase 2 Multiple-Ascending-Dose Study of SRP-5051 PPMO in Patients with DMD Amenable to Exon 51 Skipping: Part A Results
SRP-5051 is an investigational PPMO designed to skip exon 51 of the DMD gene to restore the reading frame and allow production of internally truncated dystrophin
Association of Genetic Mutations and Loss of Ambulation in Childhood-Onset Dystrophinopathy
Quantifying associations between genetic mutations and loss of ambulation (LoA) among males diagnosed with childhood-onset dystrophinopathy is important for understanding variation in disease progression and may be useful in clinical trial design.
MDA Conference Poster: Measuring Habitual Physical Activity in Duchenne Muscular Dystrophy
This is a study of the habitual physical activity of DMD patients at different stages of disease progression using actigraphy.
ACMG 2022 Conference Poster: Phase 2 Multiple Ascending-Dose Study of SRP-5051 PPMO in Patients with DMD Amenable to Exon 51 Skipping: Part A Results
A Phase 2, open-label, non-randomized, two-part dose determination and expansion study of SRP-5051 in patients with DMD.
Corticosteroids have been shown to improve muscle strength and delay loss of ambulation (LOA) in Duchenne muscular dystrophy (DMD) and are considered standard of care despite significant side-effects. The objective of this study is to evaluate whether corticosteroid treatment after LOA is beneficial for cardiac or pulmonary functions among boys with DMD.
Evaluation of effects of continued corticosteroid treatment on cardiac and pulmonary function in non-ambulatory males with Duchenne muscular dystrophy from MD STARnet
The objective of collaborative studies, such as partnering Children’s Healthcare of Atlanta, Emory University, and Rare Disease Research is to examine the prevalence of diagnoses and health disparities in our community. A study to analyze the number of boys with Duchenne muscular dystrophy (DMD) in the Atlanta area found race differences when identifying children testing positive for anti-AAV types 2 and 9Nab.
Prevalence of Anti-AAV Types 2 and 9Nab titers in Atlanta’s DMD Community
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