TOSCA – first international registry to address knowledge gaps in the natural history and management of tuberous sclerosis complex

mTOR Inhibitors in Tuberous Sclerosis Complex

(2015) Paolo Curatolo et al.   Current Neuropharmacology

Recognition of Tuberous Sclerosis in Adult Women: Delayed Presentation With Life-Threatening Consequences

(2013) Diane Seibert et al.   ANNALS OF INTERNAL MEDICINE

Everolimus treatment of refractory epilepsy in tuberous sclerosis complex

(2013) Darcy A. Krueger et al.   ANNALS OF NEUROLOGY

Lymphangioleiomyomatosis Screening in Women With Tuberous Sclerosis

(2013) Corey J. Cudzilo et al.   CHEST

Successful everolimus therapy for SEGA in pediatric patients with tuberous sclerosis complex

(2013) A. M. Cappellano et al.   CHILDS NERVOUS SYSTEM

The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia

(2013) Catherine L. Bladen et al.   HUMAN MUTATION

Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial

(2013) John J Bissler et al.   LANCET

Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma

(2013) D. A. Krueger et al.   NEUROLOGY

Are Diagnostic Criteria for Tuberous Sclerosis Still Relevant?

(2013) E. Steve Roach   PEDIATRIC NEUROLOGY

Tuberous Sclerosis Complex Surveillance and Management: Recommendations of the 2012 International Tuberous Sclerosis Complex Consensus Conference

(2013) Darcy A. Krueger et al.   PEDIATRIC NEUROLOGY

Tuberous Sclerosis Complex Diagnostic Criteria Update: Recommendations of the 2012 International Tuberous Sclerosis Complex Consensus Conference

(2013) Hope Northrup et al.   PEDIATRIC NEUROLOGY

Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial

(2012) David Neal Franz et al.   LANCET

Emerging Treatments in the Management of Tuberous Sclerosis Complex

(2012) Michael H. Kohrman   PEDIATRIC NEUROLOGY

Sirolimus Therapy for Angiomyolipoma in Tuberous Sclerosis and Sporadic Lymphangioleiomyomatosis: A Phase 2 Trial

(2011) D. M. Davies et al.   CLINICAL CANCER RESEARCH

Tuberous Sclerosis Complex: Neurological, Renal and Pulmonary Manifestations

(2011) D. N. Franz et al.   NEUROPEDIATRICS

Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis

(2011) Francis X. McCormack et al.   NEW ENGLAND JOURNAL OF MEDICINE

Everolimus for Subependymal Giant-Cell Astrocytomas in Tuberous Sclerosis

(2010) Darcy A. Krueger et al.   NEW ENGLAND JOURNAL OF MEDICINE

Registries for Robust Evidence

(2009) Nancy A. Dreyer   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

The TSC1–TSC2 complex: a molecular switchboard controlling cell growth

(2008) Jingxiang Huang et al.   BIOCHEMICAL JOURNAL

Tuberous sclerosis

(2008) Paolo Curatolo et al.   LANCET