BioMarin has provided the following update on redosing of drisapersan for a new clinical trial. Ambulation will not be an inclusion or exclusion criterion for…
BioMarin has provided the following update on redosing of drisapersen for a new clinical trial. Ambulation will not be an inclusion or exclusion criterion for enrollment, but boys will need to have been previously dosed as part of a clinical trial.
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Santhera's Raxone®/Catena® (idebenone) for the treatment of Duchenne muscular dystrophy. FDA's Fast Track program facilitates the development and review of important drugs intended to treat serious conditions and fill an unmet medical need for the purpose of getting them to the patient earlier. Santhera previously announced that the Phase III trial (DELOS) in Duchenne met its primary endpoint and demonstrated that Raxone/Catena delayed the loss of respiratory function.
I always felt a strong connection to the Duchenne community while working at the NIH. As you know, that connection and the incredible work of organizations li…
PPMD CEO, Dr. John Porter, recaps the FDA and NIH dystrophin methodology workshop. This was an important moment for the Duchenne community and PPMD was honored to be a part of it. Thank you to all the families who came to the meeting.
CAMBRIDGE, Mass. and EXTON, Pa., March 12, 2015 (GLOBE NEWSWIRE) -- Idera Pharmaceuticals, Inc. (Nasdaq:IDRA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for oncology and rare diseases, today reported its financial and operational results for the fourth quarter and year ended December 31, 2014.
United Parent Projects Muscular Dystrophy (UPPMD) requested an update from Biomarin seeking answers to specific questions around next steps in terms of re-dosi…
United Parent Projects Muscular Dystrophy (UPPMD) requested an update from Biomarin seeking answers to specific questions around next steps in terms of re-dosing, NDA submission for drisapersen in the US, the development program for additional exons, and natural history study. Read the update here.
PTC Therapeutics, Inc., today announced that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna will be made available to these siblings in advance of commercial availability in their regions, after a treating physician and the patient or guardian make a clinical decision to use Translarna, and consistent with any other applicable regulatory requirements.
PTC Therapeutics today announced that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne in advance of commercial availability in their regions.
“Families of children with Duchenne have wanted companies to be responsive in the case of siblings. With this program, PTC, once again, demonstrates itself as a champion for the Duchenne community.”
A Northbrook company said it has won fast-track status for approval of a drug that could become the first in the United States to treat Duchenne muscular dystrophy , a fatal muscle disease found most often in boys.
Earlier this month Sarepta announced a change in leadership, with CMO Ed Kaye taking over as interim CEO. Following that announcement the team at Sarepta orga…
Earlier this month Sarepta announced a change in leadership, with CMO Ed Kaye taking over as interim CEO. Following that announcement the team at Sarepta organized a call with Duchenne patient organizations and foundations to update them on the transition. Here is a short update from the team at Sarepta regarding their ongoing Duchenne program.
Sarepta Therapeutics, Inc. today announced the appointment of Edward Kaye, M.D., the company’s Chief Medical Officer (CMO), as interim Chief Executive Officer (CEO) effective immediately. He replaces Christopher Garabedian, who resigned as President and Chief Executive Officer and as a member of the Board, also effective immediately. Dr. Kaye, who will continue in a dual capacity as CEO and CMO while the company conducts a search for a new full-time CEO, will focus his efforts on heading the regulatory and clinical process for the company’s lead product candidate, eteplirsen, and follow on “exon” drug candidates for Duchenne Muscular Dystrophy (DMD).
John Porter and I have been invited to participate in the FDA-NIH dystrophin methodology meeting on Friday, March 20. While we said yes, we realize this is a…
PPMD is grateful that the FDA and NIH are willing to discuss dystrophin and have opened their doors to include our community leaders. If you would like to attend Friday’s public scientific workshop, see details here: http://1.usa.gov/1GYNSpV. If you cannot attend but would like to watch a live stream of the meeting, view this link Friday morning to attend virtually: http://1.usa.gov/1GYNSpV.
In this webinar GlobalData’s Analyst covering Immunology, Dr. Nikhilesh Sanyal, will share insights pulled from GlobalData’s Opportunity Analyzer: Duchenne Muscular Dystrophy – Opportunity Analysis and Forecast to 2019 report. Dr. Sanyal will outline and review the current DMD treatment landscape, discuss unmet needs and opportunities, and present GlobalData’s comprehensive analysis of DMD pipeline drugs. Dr. Toli Koutsokeras, GlobalData’s Senior Analyst covering Immunology, will moderate a Q&A session following the presentation.
Duchenne muscular dystrophy (DMD) represents a truly nascent market with a very strong demand for effective therapies from the patient community and clinicians. DMD is a rare and fatal genetic disorder characterized by progressive muscle degeneration, loss of ambulation, and death by the late 20s. The current standard of treatment involves generic corticosteroids, such as prednisone and deflazacort, which are used to marginally prolong muscle function and are not disease-modifying therapies.
The major obstacles that hinder the development of a clinically effective therapeutic for DMD are the multitude of DMD mutations and non-uniform progression of the disease. The two largest groups of mutations which can be targeted by a single therapeutic molecule are mutations clustered around exon-51 (approximately 13% of all DMD cases) and nonsense mutations (approximately 12.8% of all DMD cases) in the DMD gene. Hence, drug developers have to strike a delicate balance of targeting specific DMD patient populations while demonstrating sufficient clinical efficacy.
Join this webinar to increase your knowledge of the current state of the DMD market and what can be expected in this exciting therapeutic area in the future.
Liestal Switzerland March 11 2015- Santhera Pharmaceuticals (SIX: SANN) announces that Gunnar M. Buyse PhD MDProfessor of Child Neurology at the University Hospitals Leuven (Belgium) and principal investigator will present outcome data of the Phase I
Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (and were not afraid to ask) This webinar reviewed the basic biology of dystr...
The recording of our Dystrophin 101 webinar is now available. Learn more about the basic biology of dystrophin, how dystrophin is measured in both preclinical studies and clinical trials, and the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. Our speakers also discussed what is required in order to have dystrophin considered as a surrogate.
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