August 23, 2018
PTC Therapeutics has announced three Canadian sites for their clinical trial known as Study 041 for Duchenne muscular dystrophy. Study 041 is looking at how the investigational treatment, ataluren, affects ability to walk and endurance in males aged five years and older with nmDMD, compared to placebo. More information about the study can be found by clicking here […]
August 14, 2018
I am very happy to share with you today the news that Rick Moss, our Director of Advocacy and Partnerships, has joined the Board of Directors of the Canadian Organization for Rare Disorders (CORD). Over the past two years Rick has been working diligently on behalf of Jesse’s Journey in the area of advocacy with […]
August 14, 2018
Last weekend was the 15th annual Tory’s Tourney for Jesse’s Journey, hosted by the Provenzano family in Windsor. It was another sold-out tournament – over the last 15 years, this amazing event has raised more than $400,000 for Duchenne muscular dystrophy research. And they’ve never been rained out! (knock on wood). Representing Jesse’s Journey were […]
August 13, 2018
from Ana Pena
The first patient has been enrolled in a Phase 2 trial evaluating the effectiveness and safety of MNK-1411, an investigational therapy for Duchenne muscular dystrophy (DMD), announced Mallinckrodt Pharmaceuticals, the treatment’s developer. The company also reported that MNK-1411 was granted orphan medicinal product status by the European Medicines Agency, facilitating the development and eventually the approval process for the treatment candidate. The therapy was given orphan drug designation in […]
August 9, 2018
20-years-ago-today was my 122nd day on the road and I had reached London, my home town. It had been four months since I dipped the toes of my running shoes in the cold water of the Atlantic Ocean in St. John’s, Newfoundland and set out to walk across Canada at the age of 52. There’s […]
August 7, 2018
from Larry Luxner
Gavin Ward, a spunky 8-year-old Arkansas boy with Duchenne, enjoys building contraptions with his Lego set and watching video games. He also walks up to 18,000 steps a day and accompanies his father, Bruce, on outings near their home in Royal, about 70 miles west of Little Rock. But what makes Gavin stand out in […]
August 1, 2018
from American Heart Association
SAN ANTONIO, TEXAS, Aug. 1, 2018 — A protein known to drive nerve cell survival in the brain and spinal cord might also protect failing hearts in children and young adults with Duchenne muscular dystrophy, according to preliminary research presented at the American Heart Association’s Basic Cardiovascular Sciences Scientific Sessions, a premier global exchange of […]
July 30, 2018
from Ana Pena
A Phase 1/2 trial evaluating Sarepta’s microdystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD) was placed on clinical hold by the U.S. Food and Drug Administration. The FDA found trace amounts of plasmid DNA in the raw material used to make the drug. The fragments don’t seem harmful in preliminary tests and the dosing of patients by the end of […]
July 26, 2018
from Diogo Pinto
Insilico Medicine and A2A Pharmaceuticals, two leading artificial intelligence (AI) biotechs, are collaborating to create a new company called Consortium.AI, which will advance AI for the development of new molecules to treat Duchenne muscular dystrophy (DMD) and other orphan diseases. Consortium.AI will feature computational new candidates designed to target specific diseases that were validated through Insilico’s AI systems. A2A Pharmaceuticals […]
July 26, 2018
from Emma Yasinski
Discussions of the work being done on four different approaches to treating Duchenne muscular dystrophy (DMD) — repairing or replacing dystrophin, managing inflammation, improving muscle mechanics, and editing dysfunctional genes using CRISPR/CAS9 — were a highlight of sessions at the recent 2018 Parent Project Muscular Dystrophy (PPMD) conference in Scottsdale, Arizona. Patients, parents, doctors, researchers, and industry executives […]
