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TAS‐205 Safe, May Slow Muscle Loss in DMD Patients, Phase 2 Data Suggest
NewsOral treatment with TAS-205 is safe, and may delay muscle deterioration in patients with Duchenne muscular dystrophy (DMD), an ... Read more -
Addressing the Funding Crisis for Social Security
columns, Living, Learning, Thriving - a Column by Ralph YanizFor those of you who follow my column, I have some good news: Sammy Eikahldit is back. My alter ... Read more -
CureDuchenne Teams Up with Vivli to Share DMD Clinical Trial Data
NewsSeeking to improve treatment of Duchenne muscular dystrophy (DMD), CureDuchenne has teamed up with Vivli to make clinical trial data ... Read more -
FDA Accepts New Drug Application for Potential DMD Treatment Viltolarsen
NewsThe U.S. Food and Drug Administration (FDA) accepted NS Pharma‘s new drug application for viltolarsen, a potential treatment for Duchenne ... Read more -
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
NewsThe U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget ... Read more -
BIND Project Focuses on Brain Effects of Muscular Dystrophies
NewsA new project called BIND — Brain Involvement iN Dystrophinopathies — seeks to better characterize how the brain is affected ... Read more -
World Duchenne Organization Takes Lead in European Efforts to Better Treat DMD
NewsEighty delegates from 37 countries recently met in Athens for the 2019 Duchenne Patient Academy — a three-day series ... Read more -
Orsini Specialty Pharmacy Now Provides Vyondys 53 to Treat Duchenne
NewsOrsini Pharmaceutical Services is now providing Sarepta Therapeutics’ newly approved therapy Vyondys 53 (golodirsen) to treat people with Duchenne ... Read more -
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
NewsThe gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business ... Read more -
From Care Centers to Research Awards and Summer Camp, MDA Leads by Helping Others
NewsExpecting to award roughly $18 million in grants in 2020 to support research across neuromuscular diseases, the Muscular Dystrophy ... Read more -
Combo Treatments, Gene Therapies and What’s Ahead for Muscular Dystrophy: Talk with MDA
NewsGene therapies — after a tragedy kicked them back to the lab some two decades ago — are beginning ... Read more -
Blocking IGF2R Protein Could Be Therapeutic Strategy, Improve Muscle Strength in DMD, Mouse Study Suggests
NewsBlocking the protein receptor IGF2R could promote muscle regeneration and improve muscle strength in people with Duchenne muscular dystrophy ... Read more
