Our CMT Research Approach & Philosophy

We invest in the most promising Charcot-Marie-Tooth research that will best achieve treatments and a cure, and create strategic alliances within the academic, biotech, pharmaceutical and regulatory communities to accelerate progress. 

We fund projects with the highest likelihood of leading to clinical trials and approved treatments in the near term. All investments are gated, meaning projects need to achieve milestones defined for each phase of work before receiving funding for the next.

Research proposals are carefully vetted by the CMTRF’s Scientific Advisory Board.  The Board evaluates each proposal based on the excellence of the investigator, the work proposal, as well as the project’s ability to be translated into treatments for people with CMT.  Because we feel a sense of urgency to deliver important scientific breakthroughs, proposals are quickly, but comprehensively, vetted.

 We forge partnerships that include a revenue share program, which will in turn be reinvested into translational research for treatments and cures for all types of CMT. Every contract includes a return on the Research Foundation’s initial investment which is then reinvested in new projects.

The Evolution of CMT Research

The ongoing evolution of CMT research offers hope for groundbreaking advancements in the understanding and management of Charcot-Marie-Tooth disease. As researchers delve deeper into the genetic, molecular, and cellular underpinnings of CMT, the potential for developing targeted therapies increases. Current research spans a wide array of approaches, including genetic therapy aimed at correcting the underlying genetic abnormalities in various types of CMT, pharmacological studies that look to ameliorate symptoms or halt the progression of the disease, and innovative technologies like CRISPR for potential future treatments. The collaboration among global research institutions and the pooling of resources through databases and biobanks are crucial in accelerating the pace of discovery and the translation of research findings into clinically effective treatments.

A Focus On Personalized CMT Research

CMT treatment research is progressively focusing on personalized medicine approaches, recognizing the genetic diversity within the CMT community. Studies are increasingly aiming to tailor treatments based on the specific genetic mutations and clinical manifestations observed in patients. This approach ensures that therapies are not only more effective but also minimize potential side effects by aligning more closely with each patient’s unique genetic profile. Continued support and funding for CMT disease research are vital to push these initiatives forward, ensuring that the momentum in the quest for a cure and effective treatments continues to build.

A Promising Future for CMT Disease and Treatment Research

Through comprehensive research efforts, the future for those affected by CMT looks promising, with the ultimate goal of significantly improving their quality of life through scientifically backed, effective treatments.