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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
Kimonis, Virginia
NCT01166854: Characterization of Familial Myopathy and Paget Disease of Bone	Recruiting	N/A	25	US	Diffuse Optical Spectroscopy	University of California, Irvine, Beckman Laser Institute University of California Irvine	Muscle Disorder, Bone Disorder	06/22	06/22
NCT04552691: Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients	Approved for marketing	N/A		US	Pegunigalsidase Alfa	Chiesi Farmaceutici S.p.A., Chiesi USA, Inc.	Fabry Disease
NCT01353430: Characterization of Inclusion Body Myopathy Associated With Paget's Disease of Bone and Frontotemporal Dementia (IBMPFD)	Recruiting	N/A	50	US		University of California, Irvine	Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia, Paget Disease of Bone, Frontotemporal Dementia, Myopathy	12/25	12/26