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…
CAP-1002, developed by Capricor Therapeutics, safely and effectively treats cardiac anomalies associated with Duchenne muscular dystrophy (DMD). So says a clinical study whose preliminary results formed the basis of an April 28 webinar jointly hosted by Capricor and the nonprofit groups Coalition Duchenne and Parent Project Muscular Dystrophy. DMD, which occurs in…
Resolaris, a protein therapy developed for rare muscle diseases by aTyr Pharma, improved muscle strength and quality of life in a small study of patients with early onset facioscapulohumeral muscular dystrophy (FSHD). These findings, as well as others regarding the effect of Resolaris in patients with limb girdle muscular…
Japan has put Duchenne muscular dystrophy (DMD) drug candidate DS-5141b on its SAKIGAKE list, a designation to the U.S. Food and Drug Administration’s Fast Track Designation. United States’ fast tract. Daiichi Sankyo, the drug’s developer, announced the decision in a press release. An open-label Phase 1/2 clinical trial (NCT02667483), currently recruiting participants,…
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