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 […]
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 […]
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 […]
November 13, 2018
from Global News Wire
Focus will be on Milestones for Development and Commercialization CAP-1002 Therapy for Duchenne Muscular Dystrophy LOS ANGELES, Nov. 12, 2018 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR) announced today that it will meet with the U.S. Food and Drug Administration (FDA) in December to discuss clinical trial design, surrogate or intermediate endpoints and manufacturing processes for CAP-1002, […]
November 5, 2018
from Muscular Dystrophy News Today
Capricor Therapeutics’ vesicle-based therapy CAP-2003, derived from the company’s proprietary cardiosphere-derived cells (CDCs), may be an effective therapy for Duchenne muscular dystrophy (DMD), according to preclinical data. The company presented the results in August in two scientific posters at the 2018 Gordon Research Conference on Extracellular Vesicles in Newry, Maine. Capricor’s lead candidate for the treatment of DMD is CAP-1002, which is […]
October 15, 2018
from Muscular Dystrophy News Today
A new investigational therapy called GsMTx4, developed by researchers at the University of Buffalo and based on a molecule found in tarantula venom, is able to prevent the loss of muscle mass and muscle injury in a mouse model of advanced Duchenne muscular dystrophy (DMD). These findings were published in the study, “GsMTx4-D provides protection to the D2.mdx mouse,” […]
October 9, 2018
from prnewswire.com
PTC Therapeutics Announces Initial Data from Patient Registry Demonstrating Translarna™ (ataluren) Slows Disease Progression in Children with Duchenne Caused by a Nonsense Mutation SOUTH PLAINFIELD, N.J., Oct. 6, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna™ (ataluren), underscoring the long-term clinical benefit of Translarna when […]
October 4, 2018
from Matthew Herper
An experimental gene therapy dramatically improved the ability of a young boy with Duchenne muscular dystrophy to rise from the ground and climb stairs, repeating and improving on results seen in three previous patients who received the treatment, which is being developed by Sarepta Therapeutics of Cambridge, Massachusetts. “The cautionary note is this all has […]
