Duchenne Muscular Dystrophy Research
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Santhera Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne Muscular Dystrophy (DMD)

Santhera Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy Research | Scoop.it
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Summit’s IND Cleared by FDA Allowing Expansion of PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid (SMT C1100), Into the US

Summit’s IND Cleared by FDA Allowing Expansion of PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid (SMT C1100), Into the US | Duchenne Muscular Dystrophy Research | Scoop.it
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Four-Year Eteplirsen Results Show 10 of 12 Children with Duchenne Muscular Dystrophy Are Still Walking

Ten of 12 children with Duchenne muscular dystrophy (DMD) were still walking four years after treatment began with the investigational drug eteplirsen, compared with only one of 13 historical controls with DMD, researchers reported here at the AAN Annual Meeting on Tuesday.
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Catabasis Pharmaceuticals Initiates Part B of the MoveDMD Trial of CAT-1004 for Duchenne

Catabasis Pharmaceuticals Initiates Part B of the MoveDMD Trial of CAT-1004 for Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
Catabasis announced positive biomarker data from Part A of the MoveDMD trial, as well as initiation of dosing for the first patient for Part B (Phase 2) - a 1…
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Catabasis has announced positive biomarker data from Part A of the MoveDMD trial, as well as initiation of dosing for the first patient for Part B (Phase 2) - a 12-week trial to assess the efficacy of CAT-1004 in Duchenne. We look forward to continued success from our partners at Catabasis and thank you to all the families participating in this trial!
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BMS Anti-Myostatin Adnectin Program [March 2016 Webinar]

On March 30, 2016, PPMD and Bristol-Myers Squibb (BMS) hosted a webinar to discuss the BMS Anti-Myostatin Adnectin Program, recent preliminary data from th
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On March 30, 2016, PPMD and Bristol-Myers Squibb (BMS) hosted a webinar to discuss the BMS Anti-Myostatin Adnectin Program, recent preliminary data from the first-in-human study of the BMS anti-myostatin, and a clinical trial that BMS is conducting to test this drug in boys with Duchenne muscular dystrophy. Visit BMS Study Connect Patient and Care Giver Portal to subscribe for updates on the Duchenne clinical study(s) at http://bmsstudyconnect.com or visit http://clinicaltrials.gov. For any additional questions please call or email BMS at 855-907-3286 / clinical.trials@bms.com.
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Summit Reports Positive Interim Data from New Formulation of SMT C1100 in Duchenne

Summit Reports Positive Interim Data from New Formulation of SMT C1100 in Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
Summit Therapeutics announced positive interim data from a Phase 1 clinical trial evaluating a new oral formulation of its lead utrophin modulator, SMT C1100,…
ParentProjectMD's insight:
Summit Therapeutics announced positive interim data from a Phase 1 clinical trial evaluating a new oral formulation of its lead utrophin modulator, SMT C1100, for Duchenne. In this dose escalating clinical trial's initial dosing period, all patients achieved plasma drug levels within the range believed to be necessary for a potential therapeutic benefit. This Phase I trial testing the new formulation is a separate trial to the Phase 2 PhaseOut DMD trial the company has been promoting. The initial dose tested of the new formulation was one tenth of that required with the current formulation of SMT C1100 to achieve similar drug concentration levels tested in the recent Phase 1b modified diet clinical trial of SMT C1100.
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PPMD is Initial Partner in OnPAR — Online Partnership to Accelerate Research

PPMD is Initial Partner in OnPAR — Online Partnership to Accelerate Research | Duchenne Muscular Dystrophy Research | Scoop.it
The National Institutes of Health (NIH) is one of the biggest funders of medical research in the US, and projects it has funded have touched almost every Duc…
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PPMD is proud to participate in OnPar which provides access to peer-reviewed research proposals that directly relate to Duchenne research. OnPAR will provide us with an efficient way to solicit early stage and translational Duchenne research proposals, see the breadth of research in Duchenne, and gain access to novel research strategies. Projects that come to us via OnPAR will have gone through the rigorous NIH peer review process which serves as an added level of scientific scrutiny.
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CIRM Board Champions Fight Against Rare Diseases Affecting Children

CIRM Board Champions Fight Against Rare Diseases Affecting Children | Duchenne Muscular Dystrophy Research | Scoop.it
March 16, 2016  San Francisco, CA – Five fatal conditions that begin in early childhood - Duchenne muscular dystrophy, severe combined immunodeficiency, Canavan disease, Tay-Sachs and Sandhoff disease - were among nine programs awarded more than $44.5 million by the governing Board of CIRM, the California Institute for Regenerative Medicine.
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FDA_ETEPLIRSEN_LETTER_02242016.pdf

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36 scientists sent a letter to the FDA in response to the January 15 briefing documents regarding Eteplirsen
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Capricor Awarded $3.4 Million Grant from California Institute for Regenerative Medicine

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Correlation of Utrophin Levels with the Dystrophin Protein Complex and Muscle Fibre Regeneration in Duchenne and Becker Muscular Dystrophy Muscle Biopsies

Correlation of Utrophin Levels with the Dystrophin Protein Complex and Muscle Fibre Regeneration in Duchenne and Becker Muscular Dystrophy Muscle Biopsies | Duchenne Muscular Dystrophy Research | Scoop.it

Duchenne muscular dystrophy is a severe and currently incurable progressive neuromuscular condition, caused by mutations in the DMD gene that result in the inability to produce dystrophin. Lack of dystrophin leads to loss of muscle fibres and a reduction in muscle mass and function. There is evidence from dystrophin-deficient mouse models that increasing levels of utrophin at the muscle fibre sarcolemma by genetic or pharmacological means significantly reduces the muscular dystrophy pathology.

ParentProjectMD's insight:
Summit Therapeutics plc  announces the online publication on the development of imaging techniques designed to quantify utrophin protein and muscle fibre regeneration in muscle biopsies from patients with DMD and Becker muscular dystrophy ('BMD'). This collaborative research was conducted on Summit's behalf by biomarker experts, Professor Jenny Morgan, Professor Caroline Sewry and Professor Francesco Muntoni at the Institute of Child Health, University College London with financial support from the UK charity Joining Jack.
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Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD)

Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy Research | Scoop.it
NORTHBROOK, Ill., March 9, 2016 /PRNewswire-USNewswire/ -- Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with...
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Daiichi Sankyo Announces Commencement of Clinical Trials in Japan for Duchenne Muscular Dystrophy Treatment | DNA RNA and Cells | News Channels

Daiichi Sankyo Announces Commencement of Clinical Trials in Japan for Duchenne Muscular Dystrophy Treatment | DNA RNA and Cells | News Channels | Duchenne Muscular Dystrophy Research | Scoop.it
Daiichi Sankyo Announces Commencement of Clinical Trials in Japan for Duchenne Muscular Dystrophy Treatment
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A new exon skipping therapy in Japan has been administered to the first patient today. This therapy has a different chemical backbone than either of the two exon skipping products being tested in the US and skips exon 45. As of now, this trial is only happening in Japan, but PPMD will continue to follow and keep you updated.

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Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy

Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
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Marathon Pharmaceuticals Presents Analyses at AAN of a Phase III Clinical Study of Deflazacort in Patients with Duchenne Muscular Dystrophy (DMD)

Marathon Pharmaceuticals Presents Analyses at AAN of a Phase III Clinical Study of Deflazacort in Patients with Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy Research | Scoop.it

 Marathon Pharmaceuticals, a biopharmaceutical company focused solely on finding treatments for rare diseases, today announced presentations of analyses of a pivotal Phase III study of deflazacort, an investigational glucocorticoid, in patients with Duchenne Muscular Dystrophy (DMD). Results are being shared at the 68th American Academy of Neurology (AAN) Annual Meeting in Vancouver, Canada.

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NICE Recommends Translarna™ (ataluren) for the Treatment of Patients with Nonsense Mutation Duchenne Muscular Dystrophy in England (NASDAQ:PTCT)

NICE Recommends Translarna™ (ataluren) for the Treatment of Patients with Nonsense Mutation Duchenne Muscular Dystrophy in England (NASDAQ:PTCT) | Duchenne Muscular Dystrophy Research | Scoop.it
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Capricor Therapeutics Announces DSMB Recommends Continuation of HOPE-Duchenne Clinical Trial

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PPMD Community Survey about the Akashi HT-100 study

PPMD Community Survey about the Akashi HT-100 study | Duchenne Muscular Dystrophy Research | Scoop.it

If you or your child were in the Akashi HT-100 clinical trial that was recently suspended, we invite you to take our survey. It should only take 5-10 minutes and your input is extremely important. Responses are needed by April 10th.

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If you or your child were in the Akashi HT-100 clinical trial that was recently suspended, we invite you to take our survey. It should only take 5-10 minutes and your input is extremely important. Responses are needed by April 10th.
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ReveraGen BioPharma Receives $3M for Phase 2 Studies of Vamorolone in Duchenne

ReveraGen BioPharma Receives $3M for Phase 2 Studies of Vamorolone in Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
PPMD is thrilled to see the continued progress of VBP-15, now known as Vamorolone. Progress to date includes a first in-human study of healthy volunteers and…
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PPMD is thrilled to see the continued progress of VBP-15, now known as Vamorolone. Progress to date includes a first in-human study of healthy volunteers and animal data which suggests it has none of the side effects of steroids, does not impact linear growth, and does not promote insulin resistance. NIH has awarded $3 million Vamorolone to continue clinical studies in boys 4 -7 years of age. PPMD is proud to be an early funder of Vamorolone and are hopeful that this compound will one day prove to be an alternative option for steroids.
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Update on Gene Replacement/Repair Strategies for Duchenne Muscular Dystrophy

Update on Gene Replacement/Repair Strategies for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
There has been a flurry of press recently on gene therapy in Duchenne muscular dystrophy and CRISPR technology. With this barrage of information, PPMD wanted…
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There has been a flurry of press recently on gene therapy in Duchenne muscular dystrophy and CRISPR technology. With this barrage of information, PPMD wanted to take a deeper dive into gene therapy as a potential treatment for Duchenne.
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Webinar: BMS Anti-Myostatin Adnectin Program

Webinar: BMS Anti-Myostatin Adnectin Program | Duchenne Muscular Dystrophy Research | Scoop.it
Wednesday, March 30th at 1pm eastern

Please join PPMD and Bristol-Myers Squibb (BMS) as we host a webinar Wednesday, March 30th at 1pm ET to discuss the BMS A…
ParentProjectMD's insight:
Please join PPMD and Bristol-Myers Squibb (BMS) as we host a webinar Wednesday, March 30th at 1pm ET to discuss the BMS Anti-Myostatin Adnectin Program, recent preliminary data from the first-in-human study of the BMS anti-myostatin, and a clinical trial that BMS is conducting to test this drug in boys with Duchenne muscular dystrophy.
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Catabasis Pharmaceuticals Presents Positive CAT-1004 Data from Part A of the MoveDMD Trial

Catabasis Pharmaceuticals Presents Positive CAT-1004 Data from Part A of the MoveDMD Trial | Duchenne Muscular Dystrophy Research | Scoop.it
Catabasis announced positive data from Part A of the MoveDMD(SM) trial of CAT-1004 for DMD. The results support initiation of Part B of the trial, exp
ParentProjectMD's insight:
Catabasis announced positive CAT-1004 data from Part A of their MoveDMD Trial today. PPMD is happy to be partnering with Catabasis, providing travel grants to trial participants. CAT-1004 is an oral small-molecule that the Company believes has the potential to be a disease-modifying therapy for the treatment of Duchenne, regardless of the underlying dystrophin mutation.
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Gene Editing And Muscular Distrophy | KERA

Gene Editing And Muscular Distrophy | KERA | Duchenne Muscular Dystrophy Research | Scoop.it
This hour, we'll talk about the possibility of applying gene editing techniques to humans in order to stop Duchenne muscular dystrophy.
ParentProjectMD's insight:
We are so proud of Ben Dupree and his mom, Debbie, for a wonderful interview! Ben serves on our Family Advisory Committee for our DuchenneConnect PCORNet project. Listen to the full interview with Ben, his mom, and Dr. Eric Olsen of UT-Southwestern discussing gene editing in Duchenne, a topic PPMD continues to explore. Thank you Ben, Debbie, and Dr. Olsen for representing the Duchenne community!
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Jennifer Doudna: The Promise and Peril of Gene Editing

Jennifer Doudna: The Promise and Peril of Gene Editing | Duchenne Muscular Dystrophy Research | Scoop.it
Geneticist Jennifer Doudna on the controversy about Crispr-Cas9, a technique that could potentially cure genetic diseases.
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Gene Therapy Today: Notes from PPMD’s Duchenne Gene Therapy Forum

Gene Therapy Today: Notes from PPMD’s Duchenne Gene Therapy Forum | Duchenne Muscular Dystrophy Research | Scoop.it
Gene therapy.

Those two words have been tossed around for some time, starting in the late 1990s when several gene therapy trials started, but ended with fat…
ParentProjectMD's insight:
PPMD’s SVP of Research Strategy, Abby Bronson, provides an in-depth synopsis of last week’s Gene Therapy Forum hosted by PPMD. Thank you to all of the participants in this incredibly informative panel, including members from the FDA’s CBER division and Duchenne families in-town for PPMD’s Advocacy Conference. We look forward to continuing the robust discussions around the potential of gene therapy to treat Duchenne.
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