Eloxx Pharmaceuticals Announces First Patient Dosed in Phase 1b Clinical Trial
Lead molecule targets genetic nonsense mutation in rare diseasesDose escalating study to enroll 36 subjectsData expected in the fourth quarter of 2018SA
Summit Completes Initial 24 Weeks of Dosing of Ezutromid in Patients with DMD in PhaseOut DMD Cli...
Sarepta Therapeutics Announces FDA Clearance of IND for the Company’s PPMO Exon 51 Candidate, SRP...
The Investor Relations website contains information about Sarepta Therapeutics's business for stockholders, potential investors, and financial analysts.
Accelerating the Clinical Trial Process in Duchenne
Lengthy trials with variable results, based on endpoints such as the 6-minute walk test that can limit trial enrollment to ambulant individuals with Duchenne – this is today’s reality for clinical trials in this disease. But progress is being made and PPMD is proud of our role in changing the clinical trial process. Learn more about current progress that will one day make clinical trials shorter and better for participants.
Sarepta Therapeutics Signs Exclusive Global Collaboration with Duke University for Gene Editing C...
PPMD is excited to see today’s news from Sarepta Therapeutics announcing an exclusive global collaboration with Duke University for Gene Editing CRISPR/Cas9 Technology to develop new treatments for Duchenne. If you attended PPMD’s Every Single One Tour stop at Duke University late last year, you heard Dr. Charles A. Gersbach talk about the promising gene editing work his lab is doing. Thank you to Sarepta and the team at Duke for your innovation. PPMD will continue to invest in technology like CRISPR/Cas9 as part of our Gene Therapy Initiative.
Eloxx Pharmaceuticals Announces Expansion of Intellectual Property Estate for ELX-02 with Newly G...
SAN DIEGO and TEL AVIV, Israel and BOSTON, Oct. 16, 2017 /PRNewswire/ -- Sevion Therapeutics, Inc. (OTCQB:SVON) and Eloxx Pharmaceuticals Ltd.,
[Webinar] FibroGen Update on Pamrevlumab & Enrolling Non-Ambulatory Study - October 2017
PPMD and FibroGen hosted a webinar on October 25, 2017 to hear a community update on FibroGen’s development of Pamrevlumab and currently enrollin
[Webinar] MoveDMD Trial Open-Label Extension Results - October 2017
PPMD and Catabasis Pharmaceuticals hosted a webinar on October 11, 2017 to hear a community update on the results from the open-label extension of th
2017 World Muscle Society: Highlighting Progress in Duchenne
The World Muscle Society meeting is the annual gathering that brings together the world’s top neuromuscular clinicians, academics, research scientists, and industry leaders from across the globe. Topics presented here feature breakthroughs and highlights in diseases across the neuromuscular spectrum. Over the next few days, PPMD will be sharing highlights from Duchenne presentations and posters, as well as the announcements that are being released from companies in our Duchenne space in conjunction with the meeting. It is incredible to be part of this extraordinary gathering of partners who are excited to be working with our Duchenne community and like PPMD, are eager to accelerate therapy development efforts.
Catabasis Reports Positive Results from Open-Label Extension of MoveDMD Trial with Edasalonexent
Catabasis Pharmaceuticals today reported new positive efficacy results following 24 and 36 weeks of treatment with 100 mg/kg/day oral edasalonexent in the MoveDMD Phase 2 trial open-label extension. PPMD is excited to see positive effects on muscle function with edasalonexent. Please join us as we host a webinar with Catabasis on Wednesday, October 11 at 1:00 PM EDT to hear a community update on these results. More information can be found in our blog.