Rare Disease Day – Feb 28, 2014

February 28th has been designated as Rare Disease Day, a day where patient organizations around the world unite to promote awareness of the challenges, hopes and needs of those living with rare diseases. For 2014, the focus is on caregiving, and the global theme is “Join Together for Better Care.” As an advocacy organization supporting…
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National Academies Book On Clinical Trial Data Sharing

2/4/14 National Academies Book On Clinical Trial Data Sharing The National Academies Press just published a book on sharing clinical trial data - a topic of interest to each of us in the Rhabdo community. The book is available for purchase as a paperback for $ or as a free download in pdf format. You…
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NIH scientists map genetic changes that drive tumors in a common pediatric soft-tissue cancer

A Press Release from the NIH - Effort led by Javed Khan, Scientists have mapped the genetic changes that drive tumors in rhabdomyosarcoma, a pediatric soft-tissue cancer, and found that the disease is characterized by two distinct genotypes. The genetic alterations identified in this malignancy could be useful in developing targeted diagnostic tools and treatments…
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Save the Date: Rhabdomyosarcoma & Soft Tissue Sarcoma Conference

The Pablove Foundation is hosting a scientific conference on rhabdomyosarcoma & soft tissue sarcoma November 6-8, 2014 in Los Angeles. This is a by-invitation meeting for clinician-scientists and researchers from the US and abroad; however, the symposium on Saturday November 8 will be open to the public. Registration information will likely be available late Summer/early…
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A much anticipated sequencing paper on Rhabdomyosarcoma

Comprehensive Genomic Analysis of Rhabdomyosarcoma Reveals a Landscape of Alterations Affecting a Common Genetic Axis in Fusion-Positive and Fusion-Negative Tumors. Authors: Shern JF, Chen L, Chmielecki J, Wei JS, Patidar R, Rosenberg M, Ambrogio L, Auclair D, Wang J, Song YK, Tolman C, Hurd L, Liao H, Zhang S, Bogen D, Brohl AS, Sindiri S, Catchpoole…
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Tumor Cells in a Hurry: Duct Tape will do!

A New Paper by Kickuchi, Keller and Colleagues on a completely new way of thinking about what Pax3:Foxo1a does. The paper is titled: Cell-Cycle Dependent Expression of a Translocation-Mediated Fusion Oncogene Mediates Checkpoint Adaptation in Rhabdomyosarcoma. In alveolar rhabdomyosarcoma, 70-75% of tumors carry the Pax3:Foxo1a oncogene. This oncogene seems to account for a large difference…
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FDA Public Workshops on Rare Childhood Diseases

Jennifer Inman-Cartwright emailed me today saying that she sat in on a conference call with the Children's Cancer Caucus - Advocacy (CCCA) where the topic was the FDA and orphan drug issues. She forward the slide presentations which you can view and download. The first presentation was by James E. Valentine, MHS, JD Candidate at…
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CytRx Reports Highly Statistically Significant Positive Results from its Global Phase 2b Clinical Trial

CytRx, a biopharmaceutical research and development company specializing in oncology, today announced highly positive top-line efficacy results from a multicenter, randomized, open-label global Phase 2b clinical trial. The trial investigated the efficacy and safety of aldoxorubicin compared with doxorubicin in subjects with first-line metastatic, locally advanced or unresectable soft tissue sarcomas (STS). Aldoxorubicin combines the…
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Gene Sequencing Project Finds Family of Drugs with Promise for Treating Childhood Tumor

(MEMPHIS, Tenn. – December 9, 2013) Drugs that enhance a process called oxidative stress were found to kill rhabdomyosarcoma tumor cells growing in the laboratory and possibly bolstered the effectiveness of chemotherapy against this aggressive tumor of muscle and other soft tissue. The findings are the latest from the St. Jude Children's Research Hospital–Washington University…
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Important: Request from NIH/NCI for Public Input on Availability of Clinical Trial Results

Purpose With this Notice, the National Cancer Institute (NCI) announces and seeks public input on NCI’s plan to promote and ensure public availability of results from all commenced, NCI-supported clinical trials. Background NCI, a component of the National Institutes of Health (NIH), is dedicated to improving the health of Americans by conducting and funding biomedical…
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