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Update on Summit Clinical Trials

Update on Summit Clinical Trials | Duchenne Muscular Dystrophy Research | Scoop.it
Summit Therapeutics’ SMT C1100 is a small molecule drug that targets increasing the production of utrophin. Utrophin is a protein that is present in early musc…
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Summit Therapeutics provides an update on their SMT C1100 therapy – a small molecule drug that targets increasing the production of utrophin. Summit has recently been given the go-ahead for their next trial in the UK and expects to begin enrolling UK patients into their phase 2B study (“PhaseOut DMD”) very soon; enrollment in the US is planned for third quarter 2016. Eight sites across the US, UK and Europe will participate. Learn more about this trial.

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Catabasis Quarterly Newsletter

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Catabasis provides a quarterly update on their MoveDMD trial (CAT -1004). We appreciate these updates and are proud to have a been an early supporter of this promising therapy.

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Catabasis Pharmaceuticals Announces Positive Top-Line Results from Part A of the MoveDMD Trial, a Phase 1 / 2 Trial of CAT-1004 for Duchenne

Catabasis Pharmaceuticals Announces Positive Top-Line Results from Part A of the MoveDMD Trial, a Phase 1 / 2 Trial of CAT-1004 for Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
Today, Catabasis Pharmaceuticals announced positive top-line results from Part A of the MoveDMD trial, a Phase 1 / 2 trial of CAT-1004 for treating Duchenne. T…
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A Blow to Our Community – But Our Work Continues

A Blow to Our Community – But Our Work Continues | Duchenne Muscular Dystrophy Research | Scoop.it
Today our community was dealt a painful blow as the FDA issued a Complete Response letter to BioMarin regarding the company’s New Drug Application (NDA) for Ky…
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Today our community was dealt a painful blow as the FDA issued a Complete Response letter to BioMarin regarding the company’s New Drug Application (NDA) for Kyndrisa (drisapersen). 

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Akashi Therapeutics Establishes International Partnership with Grünenthal Group on HT-100 for DMD

Akashi Therapeutics Establishes International Partnership with Grünenthal Group on HT-100 for DMD | Duchenne Muscular Dystrophy Research | Scoop.it
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…
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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.

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Upcoming Webinar 1/13: HOPE-Duchenne Trial - Capricor Therapeutics

Upcoming Webinar 1/13: HOPE-Duchenne Trial - Capricor Therapeutics | Duchenne Muscular Dystrophy Research | Scoop.it
Wednesday, January 13 at 1pm eastern


Capricor Therapeutics, Inc. is developing a unique cardiac cell therapy called CAP-1002. These allogeneic cells are be…
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Please join PPMD and Capricor as we host a webinar Wednesday, January 13 at 1pm eastern to discuss the HOPE-Duchenne trial which recently opened for enrollment in the US. 

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​Acceleron to raise $150M in stock offering for muscular dystrophy studies - Boston Business Journal

​Acceleron to raise $150M in stock offering for muscular dystrophy studies - Boston Business Journal | Duchenne Muscular Dystrophy Research | Scoop.it
The Cambridge drug developer said the money will be used to bring a drug with potential to treat facioscapulohumeral as well as Duchenne muscular dystrophy to mid-stage trials this year.
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Dystrophin Mutations Affect Not Only Existing Muscle Fibers, but Satellite Cells As Well

Dystrophin Mutations Affect Not Only Existing Muscle Fibers, but Satellite Cells As Well | Duchenne Muscular Dystrophy Research | Scoop.it
A recent paper by Michael Rudnicki’s group at Ottawa Hospital Research Institute and the University of Ottawa has provided new insights into what we view as…
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A recently published paper has provided new insights into what we view as the critical targets for therapies in Duchenne.

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Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy Research | Scoop.it

Advisory Committee Meeting Scheduled for January 22, 2016

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SickKids first to remove duplicated gene using CRISPR

SickKids first to remove duplicated gene using CRISPR | Duchenne Muscular Dystrophy Research | Scoop.it
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.
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Summit Therapeutics Achieve First Milestone in Strategic Alliance with Oxford University

SUMMIT THERAPEUTICS ANNOUNCES FIRST MILESTONE ACHIEVED IN STRATEGIC ALLIANCE WITH UNIVERSITY OF OXFORD FOR FUTURE GENERATION UTROPHIN MODULATORS FOR TREATMENT OF DMD

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PhaseBio Receives FDA Orphan Drug Designation for Vasomera (PB1046) for the Treatment of Cardiomyopathy Associated With Dystrophinopathies

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.
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BioMarin Announces Data Analysis Demonstrating Consistent Efficacy of Kyndrisa™ (drisapersen) in Comparable Patients Across Three Randomized Studies (NASDAQ:BMRN)

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...
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Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen

Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen | Duchenne Muscular Dystrophy Research | Scoop.it

View printer-friendly version BACK Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen PDUFA goal date extended by standard extension period of three months to May 26, 2016 CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb.

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PDUFA goal date extended by standard extension period of three months to May 26, 2016

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Dosing and Enrollment in HT-100 Trial Suspended - Akashi RX

Dosing and Enrollment in HT-100 Trial Suspended - Akashi RX | Duchenne Muscular Dystrophy Research | Scoop.it
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...
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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.

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Summit Therapeutics Receives Regulatory Approval to Initiate PhaseOut DMD, a Phase 2 Clinical Trial of SMT C1100 in Patients With DMD

Summit Therapeutics Receives Regulatory Approval to Initiate PhaseOut DMD, a Phase 2 Clinical Trial of SMT C1100 in Patients With DMD | Duchenne Muscular Dystrophy Research | Scoop.it

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...

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PTC Completes Rolling NDA Submission to FDA and Submits Phase 3 ACT DMD Clinical Trial Results to EMA for Translarna™ (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy (NASD...

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PTC Therapeutics Outlines Business Priorities for the Development and Commercialization of Translarna™ and Progress of the Company Pipeline (NASDAQ:PTCT)

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FibroGen Enrolls First Patients in Phase 2 Clinical Study of Anti-Fibrotic Drug Candidate FG-3019 for the Treatment of Duchenne Muscular Dystrophy

FibroGen Enrolls First Patients in Phase 2 Clinical Study of Anti-Fibrotic Drug Candidate FG-3019 for the Treatment of Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
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…
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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. 

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Finding Hope in CRISPR/Cas9

Finding Hope in CRISPR/Cas9 | Duchenne Muscular Dystrophy Research | Scoop.it
Strategies that address the basic problem in Duchenne, the absence of dystrophin, are critically important to patients and families and have attracted consid…
ParentProjectMD's insight:

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.

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BioMarin Announces That FDA Has Advised it Will Not Take Action on Drisapersen New Drug Application by PDUFA Date

BioMarin Announces That FDA Has Advised it Will Not Take Action on Drisapersen New Drug Application by PDUFA Date | Duchenne Muscular Dystrophy Research | Scoop.it
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…
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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. 

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Catabasis Pharmaceuticals Completes Enrollment for Part A of the MoveDMDSM Trial, Phase 1/2 Trial of CAT-1004 for the Treatment of Duchenne Muscular Dystrophy

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ARMGO Pharma Receives FDA Orphan Drug Designation and Rare Pediatric Disease Designation for ARM210/S48168 for the Treatment of Duchenne Muscular Dystrophy

TARRYTOWN, N.Y., Dec. 9, 2015 /PRNewswire/ -- ARMGO Pharma, Inc., a clinical stage biopharmaceutical company...
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Marathon Pharmaceuticals Launches an Expanded Access Program to Provide Deflazacort to U.S. Patients with Duchenne Muscular Dystrophy

Marathon Pharmaceuticals Launches an Expanded Access Program to Provide Deflazacort to U.S. Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
Marathon Pharmaceuticals, a biopharmaceutical company that develops treatments for rare neurological diseases, today announced that, under the authori
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Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner

Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner | Duchenne Muscular Dystrophy Research | Scoop.it
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.
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PPMD has been longtime supporters of both Dr. Froehner and Dr. Whitehead.

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