TS-000833 — Psychologist Natalie Truba and Harvard Medical School’s Molly Colvin have developed a new tool to screen and measure the emotional, behavioral, learning, and social needs of pediatric patients diagnosed with Duchenne or Becker muscular dystrophy. Both muscular dystrophies are progressive, inherited disorders that display as muscle weakness that impacts the child’s ability to stand and walk. The intention of the tool is to advise and guide providers who lack direct access to mental health providers during clinic visits. This screening provides recommendations for support, guidance, or for further investigation or intervention for triaging mental, behavioral, or learning concerns.

TS-000178 — A novel mouse model for testing exon skipping therapies for DMD disease has been generated at Nationwide Children’s Hospital. This mouse model carries a duplicated exon (exon2) in the DMD gene as compared to a point mutation in the most common mdx mouse model. This unique dystrophic mouse can serve as a preclinical testing model to test various therapies that mediate exon skipping.

TS-000127 — Putative cytidine monophosphate-N-acetylneuraminic acid hydroxylase-like protein is an enzyme that in humans is encoded by the CMAH gene. A new CMAH-Deficient mouse model for DMD-related research has been created. The CMAH-Deficient mouse model mimics the human disease better than the current standard model thus providing a model for DMD that facilitate translational research to be more relevant to issues affecting the human disease. Available through Jax Labs: Jax.org Stock #017929 Stage of Development: Development is Complete Potential Applications/Markets: It can be used to study development of drugs and biologics to treat Duchenne Muscular Dystrophy, therefore its primary application is in translational research. Opportunity / Seeking: Licensing