Treatment

Treatment

A major goal of the Center is to offer treatment to patients with rare diseases. These treatments include existing drugs that can be repurposed; new antibodies and immune therapies; DNA and RNA-based drugs that alter the transcription of pathogenic genes; plus future cellular, stem cell, and gene therapies.

Our clinic will offer advanced treatments for single patients in a personalized fashion. These treatments are the ultimate in Precision Health, guided by genetics and other biochemical testing. Treatments can include, for example, the use of monoclonal antibodies or other biologicals for genetic immune disease patients; use of novel exon-skipping drugs in Duchenne muscular dystrophy; and so on. For suitable new drugs that meet the needs of are diseases, the Center will run investigator-initiated clinical trials. These may include, for example, new biologicals or inhibitors. A link to all the current clinical trials is here.

As cellular, stem cell, and gene therapies become available, the Center will coordinate and execute relevant clinical trials.