Solid Biosciences and its subsidiary, Solid GT, announced today that the FDA and the European Commission have granted Orphan Drug designations for the compa…
Solid Biosciences and its subsidiary, Solid GT, announced today that the FDA and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001, for the treatment of people with Duchenne. Solid plans to initiate clinical studies for SGT-001 in 2017.
When I first joined the Duchenne community in 2009 at Children’s National Medical Center, I remember thinking that I was walking into a critical juncture in…
So much has happened in recent weeks in the Duchenne space that it can be hard to keep up. While approvals and regulatory actions have dominated the press, there is also a lot of pre-clinical and early stage clinical research happening behind the scenes with great potential. PPMD’s Abby Bronson, SVP, Research Strategy provides a roundup of the latest Duchenne research news.
PTC Therapeutics, Inc. today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory people with nonsens…
PTC Therapeutics, Inc. today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory people with nonsense mutation Duchenne. PPMD continues to urge the FDA to provide PTC with a full review and advisory committee meeting.
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.
Catabasis provides a quarterly update on their MoveDMD trial with edasalonexent (CAT-1004) in Duchenne, highlighting the completion of enrollment for Part B of the MoveDMD trial and other recent news and events. We appreciate these updates and are proud to have a been an early supporter of this promising therapy.
PPMD is disappointed to learn that the FDA denied PTC Therapeutics' first appeal of the refuse to file letter issued in February. As we understand it, this…
PPMD is disappointed to learn that the FDA denied PTC Therapeutics' first appeal of the refuse to file letter issued in February. As we understand it, this is a process and may involve additional appeals. PPMD will be meeting with PTC to better understand the communications that transpired between the agency and the company and how we as a community can support this process moving forward.
— 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 …
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