Title |
Tuberous sclerosis complex: the past and the future
|
---|---|
Published in |
Pediatric Nephrology, December 2014
|
DOI | 10.1007/s00467-014-3027-9 |
Pubmed ID | |
Authors |
Liesbeth De Waele, Lieven Lagae, Djalila Mekahli |
Abstract |
Renal lesions represent the second most significant cause of morbidity and mortality in patients with tuberous sclerosis complex (TSC). Recent advances in the understanding of the pathophysiology of TSC have led to the exploration of new potential therapeutic targets. Clinical trials with mammalian target of rapamycin (mTOR) inhibitors have demonstrated promising results for several indications, such as renal angiomyolipoma, subependymal giant cell astrocytoma, lymphangioleiomyomatosis and facial angiofibromas. Currently, there is a scarcity of natural history data and randomized, placebo-controlled clinical trials on TSC. Recently, however, recommendations for the diagnostic criteria, surveillance, and management of TSC patients have been updated. This review focuses on these novel recommendations and highlights the need for multidisciplinary follow-up of this multi-systemic disease. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 3 | 100% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 2 | 67% |
Practitioners (doctors, other healthcare professionals) | 1 | 33% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 76 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Researcher | 10 | 13% |
Student > Master | 10 | 13% |
Student > Bachelor | 8 | 11% |
Student > Ph. D. Student | 6 | 8% |
Student > Postgraduate | 6 | 8% |
Other | 18 | 24% |
Unknown | 18 | 24% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 36 | 47% |
Agricultural and Biological Sciences | 5 | 7% |
Neuroscience | 3 | 4% |
Pharmacology, Toxicology and Pharmaceutical Science | 2 | 3% |
Veterinary Science and Veterinary Medicine | 2 | 3% |
Other | 7 | 9% |
Unknown | 21 | 28% |