News – The Foundation to Eradicate Duchenne

Tamoxifen ameliorates symptoms of Duchenne muscular dystrophy

Posted on January 15, 2013 by

Results of new study published in the American Journal of Pathology

Philadelphia, PA, January 15, 2013 – A new study has found that tamoxifen, a well-known breast cancer drug, can counteract some pathologic features in a mouse model of Duchenne muscular dystrophy (DMD). At present, no treatment is known to produce long-term improvement of the symptoms in boys with DMD, a debilitating muscular disorder that is characterized by progressive muscle wasting, respiratory and cardiac impairments, paralysis, and premature death. This study will be published in the February 2013 issue of The American Journal of Pathology.

David Sampson

PTC THERAPEUTICS ANNOUNCES EUROPEAN MEDICINES AGENCY VALIDATION

Posted on December 6, 2012 by

PTC THERAPEUTICS ANNOUNCES EUROPEAN MEDICINES AGENCY VALIDATION OF MARKETING AUTHORIZATION APPLICATION FOR ATALUREN IN DUCHENNE MUSCULAR DYSTROPHY

Enrollment for a Global Confirmatory Phase 3 Clinical Trial Planned for 1Q13

SOUTH PLAINFIELD, NJ – December 6, 2012 – PTC Therapeutics, Inc. (PTC) today announced that the European Medicines Agency (EMA) has validated a Marketing Authorization Application (MAA) seeking conditional approval for ataluren, an investigational new drug for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Validation of the MAA confirms that the submission is complete and begins the EMA’s Committee for Human Medicinal Products’ (CHMP) review process. Ataluren is the only treatment currently in clinical development targeting the cause of disease in patients with a nonsense mutation.

“Ataluren is a promising potential therapy for nonsense mutation Duchenne muscular dystrophy,” stated Dr. Thomas Voit, Medical and Scientific Director, Institut de Myologie. “PTC has developed a standard for DMD clinical trials and now the DMD community can share in the achievement of the first MAA ever filed for DMD. We appreciate PTC’s commitment to the clinical development of ataluren for this severe disorder for which only palliative treatment options currently exist.”

Jane Baj

Prosensa Announces Progress on Exon Skipping Compounds

Posted on October 23, 2012 by

Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy – £10m in milestone payments received from GlaxoSmithKline

Leiden, the Netherlands, October 23, 2012 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has selected clinical candidates for two more compounds for the treatment of Duchenne muscular dystrophy (DMD) and has been granted orphan drug designation for two additional DMD compounds in its pipeline.

Prosensa identified suitable oligonucleotide candidates for PRO052 and PRO055, designed for the skipping of exons 52 and 55 of the dystrophin gene. PRO052 and PRO055 are currently in pre-clinical development and will be moved to clinical trials as soon as possible.

College Hill Life Sciences – Anastasios Koutsos / Henry Stanley

Despite Flat Budget, NIH’s TRND Program Tries to Spark Interest in Rare and Neglected Diseases

Posted on January 31, 2012 by

With 14 approved programs in total, TRND has progressed two into clinical development.

For the past two years NIH has been trying to double its funds for developing orphan drugs. But the FY 2012 budget offered more of the same, as in the same $24 million of annual funding set aside for NIH’s Therapeutics for Rare and Neglected Diseases (TRND) program since its creation in 2009. The institute had sought $50 million for TRND this year and last.

Genetic Engineering & Biotechnology News, January 31, 2012 (Alex Philippidis)

AVI Enters Into Collaborations for the Development of Two Additional Exon-Skipping Products for Duchenne Muscular Dystrophy (DMD)

Posted on November 17, 2011 by

Collaborations Represent Key Inflection Point in AVI’s Progress in Developing Treatments for Broader DMD Population

BOTHELL, WA, Nov 15, 2011 (MARKETWIRE via COMTEX) —

KELLY GILFILLAN: Outside the Brentwood Bubble : Local family helps raise money for Duchenne MD

Posted on October 4, 2011 by

Local Family Helps Raise Money for Duchenne MD

Results of Prosensa’s Extended Phase I/II Exon-skipping Trial in Duchenne Muscular Dystrophy

Posted on September 6, 2011 by

Results of Prosensa’s Extended Phase I/II Exon-skipping Trial in Duchenne Muscular Dystrophy

ReveraGen BioPharma Selected as Awardee

Posted on August 12, 2011 by

ReveraGen BioPharma Selected as Awardee for Inaugural National Institutes of Health’s Therapeutics for Rare and Neglected Diseases Program for its Novel Dissociative Glucocorticoid Analogue

ReveraGen’s VBP15 to be developed in collaboration with NIH for treatment of Duchenne Muscular Dystrophy

Rockville, MD – ReveraGen BioPharma, Inc., a biopharmaceutical company developing alternatives to traditional steroid compounds, including dissociative glucocorticoid analogues for use in muscular dystrophy, has been selected by the National Institutes of Health’s (NIH) Therapeutics for Rare and Neglected Diseases (TRND) program, to partner on the development of a new treatment of Duchenne muscular dystrophy (DMD).

Erica Reeves, PhD