L. El Haddad

What’s new and what’s next for gene therapy in Pompe disease?

M. ElMallah, P. Kishnani, Debolina D. Biswas, 2022, Expert opinion on biological therapy.

Monitoring and Management of Respiratory Function in Pompe Disease: Current Perspectives

P. Kishnani, B. Driehuys, D. Mummy, 2023, Therapeutics and clinical risk management.

Transitional cell states sculpt tissue topology during lung regeneration.

Arvind Konkimalla, Tomokazu Souma, Zachary J. Farino, 2023, Cell stem cell.

Respiratory neuropathology in spinocerebellar ataxia type 7

Justin S. Dhindsa, Laura M. Strickland, Logan A. Pucci, 2024, JCI insight.