Clinical Trials Research

• Clinical Trials

Provides evaluation, diagnosis, and treatment of inherited leukodystrophies (including adrenomyeloneuropathy) for:

• prenatal and pregnancy counseling,
• newborns,
• children,
• adolescents, and
• adults

If families are interested, they can enroll in the Western Leukodystrophy Project, one of the largest longitudinal studies of children with leukodystrophies.

The Leukodystrophy Clinic is also a Leukodystrophy Care Network Certified Center, meeting or exceeding the Criteria for LCN Center Certification.

Josh Bonkowsky, MD, PhD

See website or contact for additional provider names and specialties. All providers may not provide all services.

Provides personalized genetic therapies and therapy development, including rapid genetic testing and research on whole genome sequencing.

Initial efforts are focused on providing answers to critically-ill infants in the newborn intensive care unit, and children with severe seizures and heart conditions.

Josh Bonkowsky, MD, PhD- Director

Provides research, patient support, community outreach, and government advocacy. Raises money for direct private funding of research. Supports individuals and communities all across the country in working together to raise awareness and funds to develop more effective treatment options for better patient outcomes. The programs aims to create a sense of hope and community so no one has to face pancreatic cancer alone.

• Specialized Information and Referral
• Clinical Trials
• Disease/Disability Information

NIDDK research creates knowledge about and treatments for diseases that are among the most chronic, costly, and consequential for patients, their families, and the Nation. Offers clinical trials, financial assistance for researchers, health information, and publications.