Our research spans a broad range of neurodegenerative, neurological, and psychiatric conditions in which the brain and nervous system are affected. These include:
- Ataxias
- Depression
- HIV related cognitive dysfunction
- Multiple sclerosis
- Lysosomal storage disorders
- Vision and eye movement disorders
- Traumatic brain injury
- The application of novel drug therapies
- Anti-psychotics and anti-depressives
- Stem cell therapies
Collectively the group’s research covers a wide spectrum of different disciplines across basic science and the delivery of clinical care. These works include:
- The investigation of disease mechanisms
- Defining brain pathology and how this informs the
- development of more effective experimental therapies
The group has particular strengths in both T1 and T2 research and has a strong translational focus in devising therapies for conditions that are underserved and are currently incurable. This is typified by the group’s world-leading research into lysosomal storage disorders (LSDs).
- These are fatal inherited disorders such as the mucopolysacchararidoses, and neuronal ceroid lipofuscinoses (or Batten disease) that kill children.
- LA BioMed is unique in having assembled a team of investigators that is focused upon bringing therapies for these profoundly disabling diseases into the clinic.
- Pioneering enzyme replacement studies for the mucopolysaccharidosis type I were successfully conducted here at LA BioMed, and recent work has led to FDA approval of the first ever treatment for a neuropathic LSD (CLN2 disease or late infantile Batten disease).
The group’s vision is to provide the same remarkable advances for more diseases that impact the brain, and to provide better care for those affected. In addition to robust federal and non-profit organization funding, the group has a strong track record of working together with partners in industry to move these studies forward to successful completion.