Liestal, Switzerland, September 13, 2016 - Santhera Pharmaceuticals (SIX: SANN) announces that additional data from the pivotal phase III trial (DELOS) demonstrating efficacy of Raxone (idebenone) on inspiratory function in patients with DMD were pub
Quercetin /ˈkwɜːrsᵻtᵻn/ is a flavonol found in many fruits, vegetables, leaves and grains. It can be used as an ingredient in supplements, beverages, or foods.…
Dr. Selsby is exploring the potential benefit of combining lisinopril (a known cardioprotector for dystrophic heart muscle used by many in our community) with quercetin (which is believed to affect heart muscle through a different pathway) to see if the two compounds in combination would be more effective than either alone. PPMD believes it will take a combination of therapies to #EndDuchenne and we applaud Dr. Selsby’s innovative approach!
Today, Summit Therapeutics plc announced positive results from a Phase 1 trial of a new formulation of ezutromid, which they are calling F6. In this trial, S…
Today, Summit Therapeutics plc announced positive results from a Phase 1 trial of a new formulation of ezutromid, which they are calling F6. In this trial, Summit showed that F6 is able to be better absorbed in patients than the current formulation of ezutromid, which they are calling F3. Because of these positive results, Summit plans to incorporate F6 into its PhaseOut DMD clinical trial alongside F3. This allows Summit to study the effect of higher concentrations of ezutromid with F6 and evaluate how its safety and efficacy compare with the F3 formulation over the 48 weeks of dosing.
If you were unable to attend PPMD's 2016 Connect Conference or if there is a session you would like to revisit, most presentations are now available on our website. Thank you to all of our incredible speakers!
We are excited to share with you presentations from PPMD’s 22nd Annual Connect Conference! If you were unable to attend this year’s Connect Conference, this is your opportunity to review some of the incredibly informative panels and discussions from this year’s meeting. We will continue to update this page over the coming weeks with slides and video content.
You’ve seen the headlines and heard the name, now find out more about CRISPR/Cas9 and what this technology could mean for Duchenne. On July 13, 2016 PPM
Last week PPMD welcomed Dr. Eric Olson from UT Southwestern Medical Center to discuss the basics of gene editing and the potential permanent correction of Duchenne mutations with CRISPR/Cas9. If you missed it live, check out the recording to learn more about this technology and what it could mean for Duchenne!
Catabasis provides a quarterly update on their MoveDMD trial (CAT -1004 / edasalonexent), highlighting the open-label extension for the MoveDMD trial and a summary of the findings to date. We appreciate these updates and are proud to have a been an early supporter of this promising therapy!
BioMarin has provided a further update to the Duchenne Community following the company's announcement of the withdrawal of the EMA marketing application and di…
BioMarin has provided a further update to the Duchenne Community following the company's announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on compounds BMN 044, BMN 045, and BMN 053.
— Deflazacort associated with improved muscle strength compared to placebo — — Deflazacort currently undergoing Priority Review by the U.S. FDA — Northbrook, Ill. – August 30, 2016 – Marathon Pharmaceuticals LLC announced the publication of pivotal Phase 3 data evaluating the investigational drug deflazacort for the treatment of Duchenne muscular dystrophy in the journal …
Pfizer Inc. (NYSE:PFE) today announced that it has acquired Bamboo Therapeutics, Inc., a privately held biotechnology company based in Chapel Hill, N.C., focused on developing gene therapies for the potential treatment of patients with certain rare diseases related to neuromuscular conditions and those affecting the central nervous system.
PTC recently participated in discussions with FDA to discuss the Refuse to File (RTF) letter issued on February 22, 2016 with respect to the company's New Dr…
Today PTC announced that they have submitted an appeal to escalate continuing discussions about the Refuse to File letter issued earlier this year with respect to the company's New Drug Application (NDA) for Translarna. We strongly believe that PTC and the families in our community that have participated for years in this trial, deserve the opportunity to share the data they have collected and their experiences with this therapy. Join us on August 1 at 11am eastern as we host a webinar with PTC to discuss next steps with the FDA.
PPMD is deeply disappointed with the FDA’s conclusion regarding Santhera's proposed subpart H (Accelerated Approval) for Raxone (Idebenone). Because individua…
PPMD is deeply disappointed with the FDA’s conclusion regarding Santhera's proposed subpart H (Accelerated Approval) for Raxone (idebenone). Because individuals in the study were not taking steroids, FDA has recommended Santhera complete the SIDEROS study on individuals on steroids and Raxone (idebenone) prior to submitting an NDA for review. For our community, this means a delay in time that we cannot afford. We are in touch with Santhera about how we can best help moving forward.
You’ve seen the headlines and heard the name, now find out more about CRISPR/Cas9 and what this technology could mean for…
You’ve seen the headlines and heard the name, now find out more about CRISPR/Cas9 and what this technology could mean for Duchenne. PPMD welcomes Dr. Eric Olson from the Department of Molecular Biology at UT Southwestern Medical Center to discuss the basics of gene editing and the potential permanent correction of Duchenne mutations with CRISPR/Cas9.
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