January 9, 2019
from EurekAlert
The gene editing technique known as CRISPR is a revolutionary approach to treating inherited diseases. However, the tool has yet to be used to effectively treat long-term, chronic conditions. A research team led by Dongsheng Duan, PhD, at the University of Missouri School of Medicine has identified and overcome a barrier in CRISPR gene editing […]
January 4, 2019
from Global News Wire
CAMBRIDGE, Mass., Jan. 03, 2019 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ:WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the planned Phase 2/3 efficacy and safety trial for its lead Duchenne muscular dystrophy (DMD) clinical program has been selected for the U.S. Food and […]
January 4, 2019
from Muscular Dystrophy News Today
Emily Crossley was a well-known TV correspondent for Britain’s Channel 4, and a presenter for CNN International’s World Business Today program. But her own world was turned upside-down the day she learned her son, Eli, had Duchenne muscular dystrophy (DMD). “The initial diagnosis was so devastating. There’s no escape. It’s absolutely relentless,” she said. “When […]
January 3, 2019
from Catabasis
— Patient Enrollment Underway in U.S. with Nine Locations Open for Enrollment — — Global Start-Up Activities Ongoing with Clinical Trial Application Approvals Received in Canada and Multiple European Countries — CAMBRIDGE, Mass., January 3, 2019 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared an update on clinical trial sites in the United […]
December 17, 2018
As 2018 comes to a close, we are taking a look back at a very special year for Jesse’s Journey. Here are just a few of the highlights: This year marked the 20th anniversary of our founder, John Davidson’s, walk across Canada. John set out on April 10, 1998 from St. John’s, Newfoundland with a […]
November 22, 2018
On November 21, Jesse’s Journey announced to donors, volunteers, and families who are facing Duchenne muscular dystrophy that as of 2018, Jesse’s Journey has granted a cumulative total of more than $10 million to research. For more than 20 years, Jesse’s Journey has been dedicated to funding research focused on Duchenne muscular dystrophy – the […]
December 27, 2018
from Muscular Dystrophy News Today
WVE-210201, an exon 51 skipping therapy, has shown positive safety and tolerability results in a Phase 1 clinical trial in boys with Duchenne muscular dystrophy (DMD), Wave Life Sciences announced. According to Wave Life Sciences, the positive safety data supports moving the therapy into a Phase 2/3 trial, planned to start in 2019, to confirm the safety profile and establish efficacy. The company […]
December 27, 2018
from Channel NewsAsia
REUTERS: Capricor Therapeutics Inc said on Wednesday it had put on hold a clinical trial of its drug to treat Duchenne muscular dystrophy (DMD), a muscle-wasting disorder, citing a safety review. The review follows a severe allergic reaction during infusion of the drug, the company said in a filing. The patient responded well to medical […]
December 7, 2018
from Nasdaq
WVE-210201 safety and tolerability profile supports Phase 2/3 clinical trial initiation in boys with DMD amenable to exon 51 skipping Delivery of interim efficacy data from ongoing open-label extension study expected in H2 2019 CAMBRIDGE, Mass., Dec. 06, 2018 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ:WVE), a biotechnology company focused on delivering transformational therapies for […]
December 3, 2018
from Muscular Dystrophy News Today
Early safety and efficacy data from a Phase 1 trial suggest that Taiho Pharmaceutical’s investigational compound TAS-205 may hold the potential to treat patients with Duchenne’s muscular dystrophy (DMD). The results were reported in the study, “A phase I study of TAS-205 in patients with Duchenne muscular dystrophy,” published in Annals of Clinical and Translational Neurology. DMD is a […]
November 28, 2018
from Business Wire
— MoveDMD Trial Data Showed Edasalonexent Was Well-Tolerated with No Safety Signals and Confirmed NF-kB Target Engagement — CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that data from the Phase 1 MoveDMD clinical trial of edasalonexent were published in the Journal of Neuromuscular Diseases, “Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-kB […]
