AAN2023: Becker and Duchenne muscledystrophy progress largely from irreversible contraction-induced injury of skeletal muscles, making the very positive interim results of an early-phase trial with a drug that prevents these injuries worth attention.
BOSTON – Becker and Duchenne muscle dystrophy progress largely from irreversible contraction-induced injury of skeletal muscles, making the very positive interim results of an early-phase trial with a drug that prevents these injuries worth attention.
In patients with BMD or DMD, a lack of dystrophin is a key pathogenic feature, according to Dr. Donovan. She explained that dystrophin in muscles connects contractile proteins to membranes and surrounding matrix. In the presence of dystrophin, muscle fibers support each other, but when this protein is absent, contraction causes injury.
“Progression depends on the degree of muscle loss,” Dr. Donovan stated, providing the rationale for moving forward with EDG-5506.In experimental studies, modulation of fast myelin provided complete protection against muscle injury while preserving its contractile function, and this translated into protection against loss of function.
The NSAA score at 6 months improved on average by about 1 point on treatment. Natural history studies of BMD predict a 1-point reduction in NSAA score over this period of time. The modest improvements from baseline in pain scores at 1 month were sustained at 6 months.
Canada Latest News, Canada Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
The 10 Most Collectible Muscle Cars Right NowWho needs modern sports cars when you have classic muscle? These 10 models define the era of automotive and pop culture history. musclecars
Read more »
FDA Fast Tracks Potential CAR T-Cell Therapy for LupusThe cellular therapy depletes CD19-positive B cells with the goal of 'resetting' the immune system to achieve durable remission.
Read more »
Who Needs Couples Therapy When You've Got a New Season of Netflix's 'The Ultimatum'Who needs couples therapy with a trained professional when Netflix's 'The Ultimatum' is here to put your relationship through a scorching hot, televised crucible?!
Read more »
GOP senators urge Biden to reverse Title 42 termination, citing estimates of migrant delugeA group of Republican senators are calling on President Biden to reverse the decision to end the Title 42 public health order next week amid fears of a new surge.
Read more »
FUBAR Sneak Preview Finds Arnold Schwarzenegger in Puppet TherapySet to hit Netflix screens on May 25th, the following sneak preview for FUBAR finds ArnoldSchwarzenegger in therapy - puppet therapy. / netflix Schwarzenegger
Read more »
Mullen Automotive stock plunges on reverse stock split announcementMullen Automotive shares plunge after reverse stock split is announced
Read more »