07 Oct
  • By Matter Now
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World Muscle Society: Day 3 Highlights

Highlights from day three at the World Muscle Society meeting in Mendoza, Argentina, provided by CureDuchnne’s scientist, Dr. Mike Kelly.  Mallinckrodt Pharmaceuticals Mallinckrodt provided an update on their Phase 2 multicenter, double-blind, placebo-controlled study, assessing the safety and efficacy of MNK-1411 in Duchenne patients. MNK-1411 is an injectable synthetic 24-amino acid peptide analogue of adrenocorticotrophic […]

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05 Oct
  • By Matter Now
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World Muscle Society: Day 2 Highlights

Highlights from day two at the World Muscle Society meeting in Mendoza, Argentina, provided by CureDuchnne’s scientist, Dr. Mike Kelly. Sarepta’s Gene Therapy Program: Sarepta provided a much-anticipated update on their dystrophin mini gene clinical study. The latest data release reinforced the positive news reported earlier for the first three patients. Today’s data — for […]

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04 Oct
  • By Matter Now
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World Muscle Society Meeting: Day 1 Highlights

Highlights from the first day at the World Muscle Society meeting in Mendoza, Argentina, provided by CureDuchnne’s scientist, Dr. Mike Kelly.  Carrier-mothers: It’s recognized that Duchenne and Becker muscular dystrophy carrier-mothers are at high risk for developing various dystrophic complications. Recent studies suggest that the incidence of cardiac and non-cardiac manifestations occurs earlier (and with […]

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17 Aug
  • By CureDuchenne User
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Santhera Pharmaceuticals now enrolling for SIDEROS Clinical Trial

Dear Friends, We are pleased to provide you with detailed information from Santhera Pharmaceuticals about an ongoing clinical trial that your son may be able to participate in. Beyond investing in early scientific research to accelerate a pathway to a cure for Duchenne, CureDuchenne is committed to providing the Duchenne community with education, help and […]

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16 Jul
  • By CureDuchenne User
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PTC Now Enrolling for Ataluren Clinical Trial

PTC Therapeutics Update on Ataluren Study 041 PTC Therapeutics is enrolling boys and young men with a nonsense mutation in a clinical trial for Ataluren.  To ensure that participants who enroll in Study 041 get the most benefit, and to ensure their safety, there are strict criteria around who is able to enter the study. […]

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28 Jun
  • By CureDuchenne User
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Report from the 2018 New Direction in Biology and Disease of Skeletal Muscle Conference

The third day of the conference focused on stem cells, new small molecule screens, poster sessions and clinical trial updates. Hirofumi Komaki (National Center of Neurology and Psychiatry, Japan) presented results of a Japanese Phase I/II dose-finding clinical study with NS-065, an exon-53 skipping drug (NS Pharma) for the treatment of Duchenne. The drug was […]

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27 Jun
  • By CureDuchenne User
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Day Two Report from the 2018 New Direction in Biology and Disease of Skeletal Muscle Conference

The second day of the conference focused on gene editing, gene therapy as well as the latest research into the development of disease biomarkers. Presentations included: Dongsheng Duan (University of Missouri) described his research groups efforts that led to the selection of an optimized micro dystrophin gene construct for systemic AAV deliver to Duchenne patients. […]

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26 Jun
  • By CureDuchenne User
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CureDuchenne’s Chief Scientific Advisor, Michael Kelly, Reports From Day One of 2018 New Direction in Biology and Disease of Skeletal Muscle Conference

CureDuchenne staff is participating in the New Direction in Biology and Disease of Skeletal Muscle Conference in New Orleans this week.  Our staff scientist, Michael Kelly, will be providing a summary of key learnings each day.  Have a question?  Leave us a comment and Michael will respond. Jane Owens (Rare Disease Research Unit, Pfizer) provided an […]

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19 Jun
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A Day To Remember

This was one of those mornings the Duchenne community will remember! Sarepta announced that the biopsies taken from the first three gene therapy patients showed, on average, over 38% dystrophin, as measured by western blot.  This is a significant amount of dystrophin, and could be a game changer for Duchenne.  Even though this is early […]

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12 Apr
  • By Debra Miller
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CureDuchenne Shares Pfizer’s Update Regarding Initiation of Gene Therapy Trial

Today, Pfizer announced that the first patient was dosed in their mini-dystrophin gene therapy trial.  This is an exciting day for the Duchenne community.  Pfizer has the experience to conduct this trial, and manufacturing capacity to see it through to success.  Of course, this is an early stage safety trial and there is a long […]

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