Before the introduction of advanced machines, the diagnosis of DMD
used to be done by slit-lamp examination with the aid of topical glycerin to dehydrate the edematous cornea.
Muscle fiber necrosis, an accidental form of cell death triggered by physical tissue damage, is an abundant phenomenon in DMD
. However, regulated forms of cell death could alternatively be involved in muscle damage development.
Polymerase chain reaction (PCR)12 provides a key component in the diagnosis of DMD
and carriers of the disease13.
The Phase IIa-extension study (VBP15-003) published online in Neurology  was a 6-month extension to the preceding Phase IIa study (VBP15-002) assessing the efficacy and safety of vamorolone in 48 boys with DMD
aged 4 to
The Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of edasalonexent in patients with DMD
. Catabasis plans to enroll approximately 125 patients ages 4 to 7 (up to 8th birthday) regardless of mutation type who have not been on steroids for at least 6 months.
- High-impact upcoming events for drugs in the DMD
space comprise topline Phase III, Phase II, and Phase I/II trial results, expected PDUFA dates for NDA/BLA, and an estimated supplemental CHMP opinion.
Now Ian is in DMD
groups on Facebook and has friends with DMD
, but he still rarely leaves the house, unless for hospital appointments.
Vertex and Exonics Therapeutics have entered into a definitive agreement under which Vertex will acquire privately held Exonics, a company focused on creating transformative gene editing therapies to repair mutations that cause DMD
and other severe neuromuscular diseases.
Scientists from the University of Texas say what they managed to achieve was "unprecedented" and could save the lives of people living with DMD
, adding: "This would hopefully prevent patients from dying of the disease."
CYTOO and Pfizer have entered a research collaboration to modify CYTOO's existing MyoScreen platform to enable its potential use as a Duchenne muscular dystrophy (DMD
) target discovery platform.
Summary: TEHRAN (FNA)- Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD
) in a large mammal, according to a new study that provides a strong indication that a lifesaving treatment may be in the pipeline.
M2 EQUITYBITES-June 22, 2018-MHRA renews EAMS scientific opinion for Santhera's Raxone in DMD