Charley'S Fund Inc

“Benjamin seckler, md (board chairman), is husband to tracy kramer seckler (board member). Earl kramer (treasurer), is father to tracy kramer seckler (board member) and kathe kramer chase (board member). Kathe kramer chase (board member), and tracy kramer seckler (board members), are sisters.”

“No review was or will be conducted.”

“Officers, directors and key employees must review the policy annually and sign a declaration that they have complied with the policy.”

“Compensation of top management and officers of the organization is developed in consideration of performance and review of relevant competitive benchmarks and submitted to finance board members for approval.”

“The organization makes its governing documents, conflict of interest policy and financial statements available to the public upon request.”

“Importantly, amplify the impact of that funding with strategic problem-solving support to both awardees and the broader dmd research community. To beat dmd, it's not enough to find the best research; we also must proactively identify and knock down hurdles that stand in the way of efficient drug development. Since inception, we have directed almost $45m in funding to these efforts, leveraging both our own funding as well as funding from other dmd patient organizations to identify and support the best science in pursuit of treatments and a cure for dmd. In 2018, we dedicated a total of $1,859,254 to our programmatic efforts. Of this, $1,522,481 was dedicated to grants and allocations and $336,773 was dedicated to other programmatic expenses.”

“In 2018 we founded the duchenne program at umass medical school, an initiative designed to advance comprehensive care and research for dmd patients in the region, across the country, and even around the world. Our support enables the establishment of a best-in-class clinical facility and program that advances our mission to stop dmd by pursuing three mutually enhancing pillars of work: providing multidisciplinary, integrative care to boys living with dmd today, hosting cutting-edge clinical trials, and conducting original research. We provided $1.3mm to umass support this initiative in 2018. The duchenne program opened its doors in july.”

“In 2018 we created the duchenne program at umass medical school. The program is powered by a unique collaboration among the medical, research, and patient communities. Founding clinic director dr. Brenda wong, host institution umass medical center and medical school, and charley's fund work jointly to ensure that the duchenne program delivers world-class results on three key goals: deliver comprehensive, integrated care to patients host cutting-edge clinical trials drive original research and discoveries to power the next wave of breakthroughs the duchenne program already in its first year is seeing more patients than any other clinic in the world. It currently hosts two active clinical trials and is in discussions to add more. It also currently has one original research project underway and is well into developing the database that will provide a key resource in further research to follow.”

“Through our impact initiative we work with partners in the field to develop their scientific programs and to identify research roadblocks and potential solutions. The impact initiative speeds and improves all research not just one particular therapy by identifying and then solving systemic problems that make drug development slower, more difficult and more costly. Currently the impact initiative focuses on three areas: 1) improving outcome measures, 2) improving preclinical methods and decision-making, and 3) improving the design, execution, and analysis of clinical trials for dmd. We developed the impact initiative because rigorous science undertaken with urgency and focus is the only way we get to the outcomes we and all duchenne families want: drugs that work, and work well. Solving these field-wide problems and improving our tools and resources is an absolute must to get results. Outcome measures: one aspect of the drug development process that concerns us is the lack of reliable outcome measures that can determine the efficacy of an intervention over a reasonably short period of time. In 2018 we committed funds to casimir, llc which was formed to address this important need by developing a series of outcome measures designed to quantify patient quality of movement using videos of boys with dmd performing daily tasks. With our support, casimir has pursued activities to develop and refine their scoring system, gather key baseline data needed to determine its reliability and concept validity, and begun engaging with the fda to ensure acceptance of this developing tool. Also, in 2018, we continued our support for electrical impedance myography (eim), collaborating with eim founder dr. Seward rutkove to assess its suitability as an outcome measure for dmd. Eim utilizes a small, hand-held device to run a high-frequency current through a muscle. It is painless, relatively inexpensive, take seconds to perform and can be used on patients of any age. With our support, dr. Rutkove has completed critical studies to confirm eim's reliability, consistency and ability to differentiate healthy muscle from diseased muscle. Preclinical methods: one major reason we have trouble translating promising research results into actual therapies is the shortcomings in how preclinical methods and models are used by dmd researchers. Our "of mice and measures" program (omam) is a collaborative, ongoing effort that brings together experts from academia and industry to improve the decision-making process for which drugs should advance to clinical testing. Clinical trials: the collaborative trajectory analysis project (ctap) is a collaborative effort among key stakeholders in the industry, academic, and patient communities to improve the way we design, execute, and analyze clinical trials in duchenne. It makes use of a growing body of dmd natural history and clinical trial data and cutting-edge biostatistical analysis approaches to improve our understanding of the natural history of duchenne and thus, how sponsors can better design trials to identify when their treatment succeeds in changing that natural history. Ctap works to provide 1) ongoing investigation and publication on key questions facing the field and impacting clinical trial design, and 2) targeted support for sponsors designing and running trials today.”