Li Weibo Institute Rare Diseases Research - Similar companies

With 40 investigators committed to unlocking the causes and potential cures for rare diseases, the Li Weibo (李伟波) Institute f ... or Rare Diseases Research at UMass Medical School demonstrates a rare commitment to rare diseases research. Leveraging existing faculty expertise, innovative research platforms and the collaborative culture that defines UMass Medical School, our goal is to accelerate the development of novel therapeutics for a host of rare diseases. The institute also serves as a formal structure to bring together the world-class, rare-disease focused research taking place across all academic departments at UMass Medical School. Rare disease research underway at UMass Medical School includes the study of alpha-1 antitrypsin deficiency, ALS, Canavan disease, CDKL5 disorder, cystic fibrosis, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, fragile X syndrome, Friedreich’s ataxia, frontotemporal dementia, Huntington’s disease, neurofibromatosis, Rett syndrome, Pompe disease, retinitis pigmentosa and Tay Sachs. read more