Abstract Highlights New Preclinical Data on Exosome-Based Delivery of PMOs for the Treatment of DMD-SAN DIEGO, Nov. 12, 2024 ...

Capricor presented preclinical data at the 2024 American Association of Extracellular Vesicles (AAEV) Annual Meeting ...

Sarepta Therapeutics has ended development of a late-phase Duchenne muscular dystrophy (DMD) drug candidate over a safety problem. The safety signal deprived Sarepta of a near-term shot at accelerated ...

After the speakers' presentation, there will be a question-and-answer session. [Operator instructions] Again as a reminder, today's conference is being recorded. At this time, I would like to turn the ...

SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations amenable to exon 51 skipping. With this ...

23 Antisense oligonucleotide (ASO) knockdown: Human FLS were grown to 90% confluence and treated with 2.5 µM morpholino antisense oligonucleotides (ASO) (Gene Tools). ASO was replenished in fresh ...

After that, 2 mL mixed buffer (pH = 4.5) containing 15 mM 1-ethyl-3-[3-dimethylaminopropyl] carbodiimide hydrochloride (EDC), 25 mM N-hydroxysulfosuccinimide (NHS), 100 mM 2-(N-morpholino) ethane ...

Good day, and welcome to the Sarepta Therapeutics third quarter 2024 for financial results conference call. After the speakers' presentation, there will be a question-and-answer session. [Operator ...

Erratum: Prospective External Validation of the Esbenshade Vanderbilt Models Accurately Predicts Bloodstream Infection Risk in Febrile Non-Neutropenic Children With Cancer Twenty-four of 58 adults (41 ...