News

February 14, 2020February 14, 2020

TAS‐205 Safe, May Slow Muscle Loss in DMD Patients, Phase 2 Data Suggest

News

Oral treatment with TAS-205 is safe, and may delay muscle deterioration in patients with Duchenne muscular dystrophy (DMD), an early Phase 2 clinical trial suggests. The findings were detailed ... Read more

February 13, 2020February 13, 2020

CureDuchenne Teams Up with Vivli to Share DMD Clinical Trial Data

News

Seeking to improve treatment of Duchenne muscular dystrophy (DMD), CureDuchenne has teamed up with Vivli to make clinical trial data available to researchers around the world. The Vivli platform, which ... Read more

February 12, 2020February 12, 2020

FDA Accepts New Drug Application for Potential DMD Treatment Viltolarsen

News

The U.S. Food and Drug Administration (FDA) accepted NS Pharma‘s new drug application for viltolarsen, a potential treatment for Duchenne muscular dystrophy (DMD) patients who are amenable to exon 53 ... Read more

February 11, 2020February 11, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

News

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 11, 2020February 11, 2020

BIND Project Focuses on Brain Effects of Muscular Dystrophies

News

A new project called BIND — Brain Involvement  iN  Dystrophinopathies — seeks to better characterize how the brain is affected in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), ... Read more

February 10, 2020February 12, 2020

World Duchenne Organization Takes Lead in European Efforts to Better Treat DMD

News

Eighty delegates from 37 countries recently met in Athens for the 2019 Duchenne Patient Academy — a three-day series of master classes aimed at deepening global understanding of Duchenne ... Read more

February 7, 2020February 7, 2020

Orsini Specialty Pharmacy Now Provides Vyondys 53 to Treat Duchenne

News

Orsini Pharmaceutical Services is now providing Sarepta Therapeutics’ newly approved therapy Vyondys 53 (golodirsen) to treat people with Duchenne muscular dystrophy (DMD) responsive to exon 53 skipping. Orsini, which ... Read more

February 6, 2020February 6, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

News

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

February 6, 2020February 6, 2020

From Care Centers to Research Awards and Summer Camp, MDA Leads by Helping Others

News

Expecting to award roughly $18 million in grants in 2020 to support research across neuromuscular diseases, the Muscular Dystrophy Association (MDA) continues to be a leader in efforts to better ... Read more

February 6, 2020February 6, 2020

Combo Treatments, Gene Therapies and What’s Ahead for Muscular Dystrophy: Talk with MDA

News

Gene therapies — after a tragedy kicked them back to the lab some two decades ago — are beginning to come into their own, making possible work in gene-targeted ... Read more

Pin It on Pinterest