Duchenne Muscular Dystrophy Research
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Nanoparticles deliver drug to mice with muscular dystrophy - Futurity

Nanoparticles deliver drug to mice with muscular dystrophy - Futurity | Duchenne Muscular Dystrophy Research | Scoop.it
When treated with nanoparticles loaded with an immunosuppressive drug, mice with muscular dystrophy showed improved strength and heart function.
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Sarepta Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date

Sarepta Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date | Duchenne Muscular Dystrophy Research | Scoop.it
The FDA has notified Sarepta that they are continuing their review of eteplirsen and will not be able to complete their work by the Prescription Drug User Fee…
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[Webinar] MissionDMD: FibroGen’s Anti-Fibrosis Program - May 2016

On May 18, 2016, PPMD and FibroGen hosted a webinar to discuss FibroGen’s investigational drug FG-3019 (which is an anti-CTGF monoclonal antibody) and th
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WAVE Life Sciences to Advance Next-Generation Nucleic Acid Therapies to Address Unmet Need in Duchenne Muscular Dystrophy

WAVE Life Sciences to Advance Next-Generation Nucleic Acid Therapies to Address Unmet Need in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
WAVE Life Sciences Ltd. (NASDAQ: WVE) today reaffirmed its commitment to advance next-generation nucleic acid therapies to address the significant unm
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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…
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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
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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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Funding Opportunities-FY15 Duchenne Muscular Dystrophy Research Program, Congressionally Directed Medical Research Programs (CDMRP), US DoD

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Molecular Therapy - A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Molecular Therapy - A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
mt is a cross-disciplinary biomedical journal devoted to publishing the most exciting advances in pharmacology and therapeutics, as they pertain to advances in translational and clinical medicine. It is recognized as one of the most prestigious journals in the field. With an impact factor of 6.825*, mt ranks in the top 4.2% of scientific journals in the latest Science Citation Index. Published monthly online and in print.
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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
ParentProjectMD's insight:
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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