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Muscular Dystrophy Association Clinical and Scientific Conference
PF-06939926 Continues to Show Safety and Efficacy in Duchenne Muscular Dystrophy
April 02, 2021
An update on the phase 1b study of Pfizer’s investigational DMD treatment was presented at the MDA 2021 conference.
IV Edaravone Reduces Chance of Death, Tracheostomy, PAV, and Hospitalization in ALS
April 01, 2021
Researchers conducted a post-hoc analysis of Study 19 to determine hazard ratios of milestone occurrences in amyotrophic lateral sclerosis.
SGT-001 Shows Improvements in Key DMD Measures, Microdystrophin Expression
March 31, 2021
Preliminary results from the IGNITE-DMD trial also showed a decreased in creatine kinase levels and improvements on NSAA and PODCI.
AT-GAA More Efficacious than Alglucosidase Alfa in Late-Onset Pompe Disease
March 27, 2021
Researchers observed improvements in motor and respiratory function and biomarkers in the investigational treatment group.
Vamorolone for Duchenne Muscular Dystrophy Shows Better Safety Profile Than Other Corticosteroids
March 26, 2021
The VBP-LTE study found no evidence of slowed linear growth, insulin resistance, or osteocalcin decreases.
Wearable Gait Technology Shows Promise in SMA and DMD
March 25, 2021
There was very good to excellent level of agreement between the instrumented insoles and the gold-standard reference system.
Risdiplam Effective in Sustained Motor Function Improvement in SMA Types 2 and 3
March 24, 2021
Two-year data from the SUNFISH study were presented at the MDA 2021 Clinical and Scientific Conference.
Switch to Deflazacort Improves Disease Progression in Patients With Dystrophinopathies
March 23, 2021
Researchers conducted a real-world study of prednisone to deflazacort switch in patients with Duchenne muscular dystrophy and Becker muscular dystrophy.
Enzyme Replacement Therapy Shows Early Improvement in Pompe Disease
March 23, 2021
Assessment of forced vital capacity with treatment of enzyme replacement therapy generally showed some initial stability followed by slow progressive decline.
Eculizumab Demonstrates Safety, No Increased Infection Rates in NMOSD or Myasthenia Gravis
March 22, 2021
The findings were consistent with the established safety profile for eculizumab in other non-neurologic indications, according to study authors.