Solid Biosciences’ SGT-001 improved muscle activity in dogs with Duchenne muscular dystrophy (DMD), according to research the company presented at a Washington conference. Another finding of the two preclinical-trial studies was that SGT-001 increased the expression of microdystrophin, a form of the protein dystrophin found in muscle. Too little of…
Using glucocorticoid steroids once a week instead of once a day to slow the progression of muscular dystrophy (MD) could prevent downsides of the treatment, including accelerated muscle wasting, a study reports. Researchers at the Feinberg School of Medicine at Northwestern University discovered that mice with Duchenne muscular dystrophy fared better…
Acceleron Pharma said the first patient has been dosed in a Phase 2 clinical trial of ACE-083, its proprietary lead candidate drug ACE-083 to treat facioscapulohumeral muscular dystrophy (FSHD) — a genetic disease affecting 19,000 Americans for which no approved therapy yet exists. “ACE-083 has generated a strong data set in…
PTC Therapeutics announced that Emflaza (deflazacort), an approved treatment for all Duchenne muscular dystrophy (DMD) patients regardless of mutation, will be available in a matter of weeks in the U.S., and this time around with a starting list price of $35,000 a year — for a child weighing 25 kilograms, or about…
PerkinElmer has developed an improved screening method to test whether newborns may have Duchenne muscular dystrophy (DMD). The test detects the levels of a specific form of a protein called creatine kinase (CK), a disease biomarker, using dried blood spots from a baby’s blood sample. A report on these findings, “Characterization…
Andrea Dalzell, Ms. Wheelchair New York 2015, is featured among the disability advocates who will appear at the United Spinal Association’s 6th Annual Roll on Capitol Hill. Advocates will speak directly with legislators on issues affecting independence and quality of life for members of the the…
Finding out your child has a neuromuscular disorder is an incredibly difficult time for the whole family. It’s perfectly normal to feel a range of emotions, from anger to guilt, depression to denial, as you come to terms with what the future holds. Many parents will go through a period…
One of a class of compounds that GTx is developing increased the survival of mice with Duchenne Muscular Dystrophy (DMD), and improved their muscle, heart and lung function, according to a pre-clinical trial study. The therapies are known as selective androgen receptor modulators, or SARMs. The study, “Androgen Receptor Agonists…
The 7th Annual Getzlaf Golf Shootout to benefit CureDuchenne will take place Sept. 9 at Monarch Beach Golf Links in Dana Point, California. Brian Hayward will present the event, which will be hosted by the captain of the Anaheim Ducks hockey team, Ryan Getzlaf, and…
The journal Orphanet Journal of Rare Diseases has published a review highlighting the prevalence of Duchenne muscular dystrophy (DMD) as well as the disease’s costs and treatment patterns in North America and Europe. The analysis, “The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence…
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