PPMD supported the development of the HT-100 program from early on, including a $500,000 investment via our GIFTED program for the clinical trial. We are excit…
PPMD supported the development of the HT-100 program from early on, including a $500,000 investment via our GIFTED program for the clinical trial. We are excited for the expansion of the clinical trial and thrilled for the relationship with Grunenthal. This incredible partnership enables Akashi to continue to be managed and led by parents and we are all hopeful HT-100 will be part of the cocktail of combined therapies that will achieve our highest priority--to stop progression, to end Duchenne.
CRISPR is being called a game changer. A new study led by The Hospital for Sick Children (SickKids) shows how researchers used CRISPR to remove a duplicated gene from a genome. This is the first time this has ever been done.
MALVERN, Pa., Dec. 1, 2015 (GLOBE NEWSWIRE) -- PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company and leader in the field of biopolymer-based drugs, focused on developing treatments for metabolic and specialty cardiopulmonary disorders, today announced that the U.S.
SAN RAFAEL, Calif., Nov. 20, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced data analysis for Kyndrisa demonstrating consistent evidence of efficacy in comparable patients across three randomized, placebo-cont...
Dosing and Enrollment in HT-100 Trial Suspended Dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne muscular...
We are distraught to learn that dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne, are being suspended. According to what Akashi Therapeutics is reporting, one of the patients in the trial, receiving 60μg/kg/d (the highest dose in the study), is experiencing serious, life-threatening health issues, and the company is working with the FDA to analyze the situation. They do not yet know to what extent the patient’s health issues are related to HT-100 and/or to other factors.
According to the company, “Our intention is to restart the trial once our investigation into the causes of this patient’s experiences is complete, and Akashi and the FDA are satisfied that any measures that might be necessary can be put in place to address these causes.“
PPMD will continue to keep you updated on this situation as information becomes available. Please read the full statement from Akashi that answers some of the many questions we all have.
While this temporary trial suspension is difficult news for all of us, we are grateful that Akashi holds the safety of our patient community as their first priority. Our community’s thoughts and prayers are with the patient and family experiencing the serious health complication.
OXFORD, UK, Jan. 21, 2016 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (LSE:SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficile infection, announces...
FibroGen announced enrollment of their first patients in an open-label, multicenter Phase 2 clinical trial of the investigational compound FG-3019 in non-ambul…
FibroGen announced enrollment of their first patients in an open-label, multicenter Phase 2 clinical trial of the investigational compound FG-3019 in non-ambulatory patients with Duchenne. We are thankful that FibroGen is addressing ways to treat fibrosis caused by Duchenne’s progression and are hopeful that this study will prove promising.
Strategies that address the basic problem in Duchenne, the absence of dystrophin, are critically important to patients and families and have attracted consid…
Duchenne made national headlines over the holidays thanks to the latest news on gene editing technology called CRISPR/Cas9. As you heard throughout the month of December, PPMD has a long history of supporting early-stage research at pivotal moments. PPMD takes a deeper dive into gene editing – a very promising technology with the potential to end Duchenne.
BioMarin has announced that the FDA will not make a decision regarding drisapersen until early January 2016. PPMD will continue to help both BioMarin and the…
BioMarin has announced that the FDA will not make a decision regarding drisapersen until early January 2016. PPMD will continue to help both BioMarin and the FDA any way we can over the next few weeks. Thank you to the families who are spreading awareness about their experiences on drug. We will keep you updated on all developments.
In addition to their well established cholesterol lowering benefits, statins also have potent anti-inflammatory, anti-fibrotic and antioxidant effects, which continue to be identified in a wide range of diseases.
PPMD has been longtime supporters of both Dr. Froehner and Dr. Whitehead.
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