Muscular Dystrophy Association Clinical and Scientific Conference (MDA)

Over a 2-year period, SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores.

A greater proportion of patients identified by newborn screening achieved a greater than 4-point increase in CHOP-INTEND scores during the observation period compared to those clinically diagnosed.

Researchers found that physical activity as measured by wearable devices was significantly associated with in-person measures of strength and function in patients with CMT.

Most treatment-emergent adverse events were mild in severity across both studies, with no evidence of renal toxicity, cardiac signals, or deaths.

Researchers conducted a pooled safety analysis of the FIREFISH, SUNFISH, and JEWELFISH studies.

Scholar Rock noted that the topline results from the 52-week treatment period are expected to be announced in the second quarter of 2021.

LT-001 and LT-002 followed up patients treated with onasemnogene abeparvovec from the START, STRONG, STR1VE, and STR1VE-EU studies.

The executive vice president and chief research officer of the MDA offered her perspective on the ongoing conversations about and impact of the pandemic on the care of patients with muscular dystrophies.

At each timepoint, all assessed participants with 3 SMN2 copies and the majority with 2 SMN2 copies were identified by PASA as not being tube fed.

Treatment with nusinersen demonstrated no clinically meaningful changes in vital signs or clinical laboratory parameters, including urine parameters, in those with spinal muscular atrophy.

Researchers also found that, in comparing ataluren to standard care, ataluren delayed loss in pulmonary function.

The high-dose cohort in part 1 of the FIREFISH study also showed that more than 50% of infants could sit without support and more than 90% could feed orally.

Sharon Hesterlee, PhD, executive vice president and chief research officer, MDA, shares an early look at the MDA's 2021 Virtual Clincial and Scientific Conference, March 15-18.

The executive vice president and chief research officer of the MDA gave an overview of the upcoming virtual annual meeting and her insight on what to look for.