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 more than $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 2019, we dedicated a total of $1,837,658 to our programmatic efforts. Of this, $1,511,340 was dedicated to grants and allocations, and $326,318 was dedicated to other programmatic expenses.”

“We continued our support of the duchenne program at umass medical school, which we created in 2018. 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 - the largest dedicated duchenne clinic in the northeast us - delivers world-class results on three key goals: 1) deliver comprehensive, integrated care to patients; 2) host cutting-edge clinical trials; and 3) drive original research and discoveries to power the next wave of breakthroughs.the duchenne program, now in its second year, has more than 300 patients in its direct care and continues to benefit patients worldwide even if they do not enter its doors in person through sponsored clinical trials, original research, and sharing best practices in research and care with the broader community. This year, in addition to a roster of industry-sponsored clinical trials, the clinic secured irb approval for ongoing registry work and launched a biorepository.”

“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 2019 casimir, llc continued its work 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. The fda continues to be supportive and recently 'renewed' its endorsement for casimir to continue work with an fda-adjacent advisory body that provides hands-on input during development. Also in 2019, 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, takes 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. Our publication with the results of the most recent multi-site study has been published in annals of clinical and translational neurology. Charley's fund also continues to explore a potential clinical study to obtain key outstanding data in a more efficient, effective manner than two separate approaches, mentioned above. Both programs are at a point where the critical need is data to help answer a key question: "how accurately do these measures detect treatment effect?" since steroid treatment is the only approved intervention known to alter disease progression, in 2019 we started conversations with both the casimir and eim teams to consider an observational study to evaluate the two exploratory measures' sensitivity to change in disease upon initiation of steroid treatment. Preclinical methods one major reason we have trouble translating promising research results into actual therapies is the shortcomings of how preclinical methods and modules 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. To this purpose, in 2019 we committed $302,646 to leiden university medical center in the netherlands and universita degli studi di bari in italy for a large natural history study to better characterize key dmd models. A key component of the study is to unearth discrepancies in the processes different labs undertake and then publicize the best methodology to enable reliable, consistent results. Clinical trials the collaborative trajectory analysis project (ctap) is a collaborative effort among key stakeholders in the industry, academic,”