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Recent Research News

Parent Project Muscular Dystrophy (PPMD) understands how important it is for the Duchenne community to have first hand access to this research, important findings, and any other research updates that come along. Because of this, PPMD monitors and posts all current Duchenne research.

08/26/10: A recent article in Slate addresses the difficulties of developing new therapies, and the importance of the words that we all use in talking about therapy development efforts. Read the article.

08/25/10: NIH presents new funding opportunity for investigators. Check out these grants with fall application deadlines.

08/19/10: BioMarin to Host Conference Call to Discuss Results of Phase 1 Clinical Study BMN 195 for Duchenne Muscular Dystrophy. Learn more.

08/19/10: ACE-031 receives orphan designation in the US. Read the release.

08/17/10: Santhera obtains European patent for use of Catena/Sovrima in the treatment of muscular dystrophies. Read release.

08/13/10: Clinical development program update: A Phase II, double blind, exploratory, parallel-group, placebo controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability, and pharmacokinetics in ambulant subjects with Duchenne. Read more.

08/09/10: PPMD awards $600K to Nationwide Children’s Hospital for gene therapy study. Dr. Jerry Mendell leading follistatin gene therapy. Read more.

08/04/10: Acceleron Pharma receives FDA fast track designation for ACE-031 for treatment of Duchenne. Read the release.

07/20/10: PPMD awards $20,000 grant to Prothelia. Learn more.

07/14/10: PPMD announces grants for clinical study sites. The funds will help offset travel costs for participants in Acceleron Pharma’s Phase 2 clinical study of ACE-031. Read more.

07/01/10: New peer-reviewed study concludes that Protandim improves markers of oxidative stress and fibrosis in muscular dystrophy mice. Read the release.

07/01/10: Exon Skipping and Duchenne Muscular Dystrophy Therapy: Selection of the Most Active U1 snRNA-Antisense Able to Induce Dystrophin Exon 51 Skipping Read more.

06/23/10: Prosensa and GlaxoSmithKline initiate development of four additional products under existing alliance in Duchenne. Read the release.

06/18/10: A study to assess the pharmacokinetics, safety, and tolerability of GSK2402968 in non-ambulant subjects with Duchenne, is now recruiting. Drug designed to induce skipping of exon 51. More info.

06/17/10: How does the FDA make its decisions? Who makes the rules for drug development? What special accommodations are made for rare diseases? PPMD & Cure Duchenne host a two-part live webinar series with FDA officials in July. Click here for more details.

06/04/10: FDA and NIH will co-sponsor a meeting titled Antisense Oligonucleotide (AON) Therapies in Neuromuscular Diseases, taking place on September 27 and 28, 2010 in Washington DC. PPMD will participate in this important meeting. Read more.

06/03/10: Costa Rica has ordered the country's largest stem cell clinic to stop offering treatment, saying there is no proof that it is effective. Read more.

06/02/10: AVI-4658 demonstrates first ever reported generation of greater than 50% dystrophin-positive muscle fibers in a patient following systemic administration in Duchenne. Read the release.

05/28/10: PPMD President Pat Furlong discusses yesterday's open conference call with PTC Therapeutics regarding the ataluren trials. Read her latest blog.

05/27/10: Watch a video piece from the University of Florida about Dr. Krista Vandenborne who recently received NIH funding for Duchenne research, thanks in part to an End Duchenne Grant from PPMD.

05/26/10: A new clinical trial for the investigational compound GSK2402968 (formerly PRO051) was posted to www.clinicaltrials.gov on 5/24. This study will assess the pharmacokinetics, safety, and tolerability of the compound in non-ambulatory boys with Duchenne who have a dystrophin gene mutation amenable to an exon 51 skip. Visit DuchenneConnect for updates.

05/24/10: Please join PPMD on our follow-up, open conference call with PTC Therapeutics regarding the ataluren trials on Thursday, May 27 at 5pm eastern. For more information, click here.

05/18/10: Check out this important article by PPMD President Pat Furlong and Barbara Godlew, highlighting how advocacy organizations function as a direct conduit to specialized patient populations and how these groups can provide a communication channel to reach thousands of potential clinical trial participants.

05/11/10: PPMD will host a follow up call with PTC Therapeutics regarding the ataluren trials on May 27, 2010 at 5pm eastern. For more information, click here.

05/07/10: PPMD End Duchenne Grant leads to $7.5 million NIH award for University of Florida’s Krista Vandenborne, P.T., Ph.D. for her work with MRI and MRS technology. Read the release.

05/07/10: PPMD's Senior Director of Research, Sharon Hesterlee, has been traveling nonstop the last week to AVI in Seattle, the BIO meeting in Chicago, and New Directions meeting in Ottawa. Read her blog about trends in biotech and the latest on exon skipping.

04/22/10: Informative video about the technique of exon-skipping, how it works and how it’s being applied in clinical testing. The technology was developed, in part, by Steve Wilton, a regular presenter at PPMD’s Connect Conference.

04/20/10: Responding to concern within the community regarding the potential for an immune response to exon skipping, Annemieke Aartsma and Hidde Ploegh provided their thoughts. Read more.

04/16/10: Brenda Wong presented top-line results from the Phase 2b clinical trial of ataluren in patients with nonsense mutation DBMD at the American Academy of Neurology (AAN) annual meeting today. Read the summary.

04/09/10: FDA publishes update on Duchenne drug development and regulatory considerations, including findings from a study held during PPMD’s Annual Connect Conference last year.

04/08/10: If you missed today’s conference call with John Crowley, listen to it online now. John was very open about his own clinical trial experiences and comments on the recent ataluren trials. This was an incredible hour with a parent who has shared a very similar journey with many of us.

04/01/10: PPMD is thrilled to welcome John Crowley, CEO and President of Amicus Therapeutics, inspiration for the movie “Extraordinary Measures,” for a conference call about his own experiences with the clinical trial process on April 8 at 1pm EST. Read more.

04/01/10: Family friendly version of Care Considerations available from TREAT-NMD. Download here.

03/30/10: Prosensa initiates clinical development of PRO044 in an open label phase I/II study. Read more.

03/30/10: The American Academy of Neurology’s annual meeting will be April 12-17, 2010. PPMD will be attending and will provide reports to the community. On the agenda, updates and results of clinical trials on ataluren as well as exon skipping. Until then, check out the latest AVI update expected to be discussed at this meeting.

03/26/10: PTC Therapeutics has responded to questions you submitted regarding the ataluren trial. Click here to read their summary of answers. Additionally, Genzyme has issued a joint statement with PTC to the community. Read this message.

03/25/10: AVI BioPharma announces update on AVI-5038, its PPMO Duchenne drug candidate, to be presented April 14, 2010 at the American Academy of Neurology Annual Meeting. Read more.

03/24/10: Spanish translation of the notes from the ataluren call with PTC is now available.

03/22/10: Advocacy in Action: Jackson Laboratory receives $1.44m federal grant for Duchenne models. Learn more.

03/19/10: In response to some of your questions about the ataluren survey, PPMD has posted FAQs. And if your child took part in the ataluren trial, and you have not participated in the survey, please do so today.

03/18/10: Researchers discover chemical that may protect hearts of muscular dystrophy patients. Read more.

03/16/10: If your child took part in the ataluren trial, we want to hear from you. Click here to help us gather important data.

03/12/10: FDA Officials go to new length to encourage applications for Orphan-Drug Status. Read more.

03/08/10: In response to the press release that went out on March 3, 2010 from PTC Therapeutics regarding the ataluren trials, an open conference call was held on 3/5 for parents to have their questions answered by PTC. Click here to read notes compiled from this important open conference call.

03/05/10: PPMD President Pat Furlong talks about the ataluren trial results as it relates to the community, in her latest blog.

03/05/10: PPMD Board Chairman, John Killian, shares his thoughts after Friday's call with PTC. Click here to read.

03/04/10: Important open conference call tomorrow regarding the results of the ataluren trial. Click here for call-in information.

03/03/10: Ataluren results announced. Read this important statement from PPMD President, Pat Furlong.

02/18/10: AVI BioPharma receives grants totaling $500,000 to support continuing development of drug candidates to treat Duchenne. Read more.

02/09/10: Günter Scheuerbrandt recaps the TREAT-NMD and NIH conference held last November in Belgium. Learn more about the presentations from this important conference in neuromuscular disease. And if you missed PPMD President Pat Furlong’s blog about the conference, check it out.

TREAT-NMD’s website helps distinguish between the hype of stem cell research and the hope that this science may bring to diseases including Duchenne. (02/05/10)

Parents: Help in an effort to reduce the time to diagnosis! Take a survey about your first concerns for your child with Duchenne. (01/26/10)

BioMarin initiates Phase 1 Clinical Study of BMN 195 for Duchenne. (01/26/10)

Important revision to Part 2 of Care Considerations. Please download this version; it should replace any previous version. Change takes place on page 185 where indicated, with correction on page 189. (01/21/10)

PBS TV Special Rare disease diagnosis through technology. Read more about this upcoming TV special. (01/20/10)

PTC Therapeutics announces additional study of ataluren in patients with advanced nonsense mutation Duchenne/Becker. (01/19/10)

BioMarin initiates Phase 1 Clinical Study of BMN 195 for Duchenne. (01/13/10)

Research could predict age of onset of heart problems in Becker muscular dystrophy. (01/13/10)

Systemic treatment with AVI-4658 demonstrates RNA exon skipping and dystrophin protein expression in Duchenne patients. (12/23/09)

PTC gets $50M to launch Ataluren, further studies. (12/23/09)

PTC opens ataluren trial to Non-ambulatory Patients With Nonsense-Mutation-Mediated. Learn more and check out Pat Furlong’s thoughts. (12/11/09)

CDC strongly recommends that any close family members and caregivers of someone diagnosed with a neuromuscular disease should receive the H1N1 vaccine. (12/10/09)

Major medical publication supports 6-Minute walk test, funded in part by PPMD. (12/08/09)

Lancet Neurology publishes CDC’s Care Considerations after three years of vetting and development. Read this critical document, that provides important information regarding the care of your son. (12/04/09)

What are the correlations between cognitive impairments and late deletions? A recent report published in HMG Advance Access explains. (11/20/09)

PPMD President Pat Furlong most recent update from Treat-NMD in Brussels. (11/19/09)

PPMD President Pat Furlong reports from Treat-NMD in Brussels. (11/17/09)

Latest issue of Nature Biotechnology weighs in on the business of stem cells. (11/17/09)

Meet The Pediatric Neuromuscular Clinic at MassGeneral Hospital for Children. (11/12/09)

PPMD President, Pat Furlong, weighs in on stem cell research report from Costa Rica (11/10/09)

PPMD awards UCLA latest end Duchenne grant. (11/09/09)

UPPMD hosts conference on “The Endocrine Aspects of Duchenne Muscular Dystrophy” in Florence later this week. Learn more about the agenda. (10/27/09)

Check out three recently published articles about exon skipping: 1. From Lancet Neurol 2. From The Journal of Gene Medicine 3. From BMC Medical Genetics (10/27/09)

Exon Skipping Drug Prevents Muscle Wasting and Maintains Muscle Function in Severely Affected, Dystrophin Deficient Mice. Read the press release and article. (10/23/09)

PPMD adds name to letter to Congress in support of the Therapeutic Discovery Project Tax Credit. (10/22/09)

National Health Counsel analyzes potential health care reform and breaks down what it may mean for you as a patient. (10/20/09)

Cure for Duchenne Lies in Paws of Man's Best Friend – Interview with Joe Kornegay (10/16/09)

MD STARnet Reports Prevelance of Duchenne Among Males Age 5-24 (10/15/09)

AMT Receives EMEA Orphan Drug Designation for Duchenne Muscular Dystrophy (10/14/09)

Prosensa and GlaxoSmithKline form alliance to fight Duchenne. Read the official press release. (10/13/09)

With H1N1 vaccination questions all over the news, you may be confused about what is best for your child. Dr. Brenda Wong of CCHMC weighs in. (10/13/09)

CDC releases important brochure about H1N1 and what you can do to prevent your family from getting infected (10/08/09)

TREAT-NMD wraps up promising, successful workshop. Read the press release (10/02/09)

PPMD President Pat Furlong recounts her recent trip to London for the TREAT-NMD workshop on exon skipping. (09/29/09)

CDC and PPMD Complete Duchenne Fact Sheet (09/29/09)

TREAT‐NMD Workshop re: the Development of Antisense Oligonucleotide Therapies for Duchenne. Read the conference outline and agenda. (09/25/09)