Wednesday 21 January 2015

» Marathon Pharmaceuticals announce FDA fast track designation for deflazacourt Action Duchenne

» Marathon Pharmaceuticals announce FDA fast track designation for deflazacourt Action Duchenne: "Marathon Pharmaceuticals, LLC, a biopharmaceutical company focused on developing treatments for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for deflazacort in the treatment of patients with Duchenne Muscular Dystrophy (DMD). Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties."



'via Blog this'

Heart Failure Drugs Slow Deadly Damage in Duchenne, Offering New Hope - PPMD Community

Heart Failure Drugs Slow Deadly Damage in Duchenne, Offering New Hope - PPMD Community: "Less heart muscle damage was seen at 6 months, and less decline in heart function was seen at 12 months when adding eplerenone to background ACE inhibitor or ARB therapy compared to adding placebo. The research team believes that by benefiting heart function at a very early stage, longer-term complications of cardiomyopathy such as heart failure can be greatly reduced. While continued research is needed to develop even better treatments for tomorrow, the availability of these medicines offers boys with Duchenne something beneficial for their hearts today."



'via Blog this'

Update on the 2015 Flu - PPMD Community

Update on the 2015 Flu - PPMD Community: "What should I do if I think my child has the flu?
 
If your child has symptoms of the flu, call your primary health care provider immediately!  Rapid diagnostic testing, which checks secretions in the throat for the influenza virus, is 50-70% accurate for diagnosing influenza (most providers will send a negative specimen for culture, just to be sure that there is no virus present).  If the test is positive, starting an antiviral medication is recommended.  Antiviral medication should be started as soon as possible in the course of the illness and continued for 5 days.   It is best if this medication is started within 48 hours of the beginning of symptoms, but the treatment can still has some benefit if started later, especially for patients who are experiencing a longer or more complicated course."



'via Blog this'

Sunday 11 January 2015

Tivorsan Pharma Meets Development Milestones with DMD Treatment Research

Tivorsan Pharma Meets Development Milestones with DMD Treatment Research: "Tivorsan Pharmaceuticals, Inc. (Tivorsan), a protein therapeutics company with a focus on developing treatments for neuromuscular disorders such as muscular dystrophy, recently announced the company has achieved a fourth set of pivotal milestones under a series of research grants from Parent Project Muscular Dystrophy (PPMD) and Muscular Dystrophy Association (MDA). These achievements mark the release of more funding from these grants, in fulfillment of PPMD’s pledge of $565,000, and the MDA’s pledged $1 million."



'via Blog this'

Friday 9 January 2015

Tivorsan Pharmaceuticals Achieves Key Development Milestones under its Parent Project Muscular Dystrophy and Muscular Dystrophy Association Awards | Business Wire

Tivorsan Pharmaceuticals Achieves Key Development Milestones under its Parent Project Muscular Dystrophy and Muscular Dystrophy Association Awards | Business Wire: "PROVIDENCE, R.I.--(BUSINESS WIRE)--Tivorsan Pharmaceuticals, Inc. (Tivorsan) today announced that it met its fourth (4th) set of critical milestones under its existing grants from Parent Project Muscular Dystrophy (PPMD) and Muscular Dystrophy Association (MDA). The achievement of these milestones triggers additional payments to Tivorsan pursuant to the Company’s $565,000 grant from PPMD and $1,000,000 grant from MDA."



'via Blog this'

Thursday 1 January 2015

Early use of heart failure drugs slows cardiac damage in boys, young men with DMD

Early use of heart failure drugs slows cardiac damage in boys, young men with DMD: "Early use of available heart failure drugs slows the progressive decline in heart function before symptoms are apparent in boys and young men with Duchenne muscular dystrophy (DMD), according to a new study published online by The Lancet Neurology."



'via Blog this'