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Assessing the effectiveness of rapamycin on angiomyolipoma in tuberous sclerosis: a two years trial
Tuberous sclerosis (TS) is a rare autosomal dominant systemic disease with an estimated prevalence of 1/6000. Renal angiomyolipoma (AML) is a benign tumour with high morbidity frequently present in TS. The aim of the study was to test the effect of rapamycin in reducing the volume of AML in TS. Twen...
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| Published in: | Orphanet journal of rare diseases 2012-11, Vol.7 (1), p.87-87, Article 87 |
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| creator | Cabrera-López, Cristina Martí, Teresa Catalá, Violeta Torres, Ferran Mateu, Silvia Ballarín, Jose Torra, Roser |
| description | Tuberous sclerosis (TS) is a rare autosomal dominant systemic disease with an estimated prevalence of 1/6000. Renal angiomyolipoma (AML) is a benign tumour with high morbidity frequently present in TS. The aim of the study was to test the effect of rapamycin in reducing the volume of AML in TS.
Twenty four-month prospective open-label, single arm, unicentre Phases II andIII study. The primary endpoint was to evaluate the effect of treatment on the reduction of at least 50% AML volume from baseline at 24 months. The secondary endpoints were: average tumour reduction, surgical complications, skin lesions and drug safety.The study population comprised 17 patients, aged >10 years who were diagnosed with TS and had ≥1 renal AML >2 cm of diameter and had a serum creatinine < 2mg/dl and urine protein/creatinine ratio < 22.6 mg/mmol. The trial was conducted at Fundació Puigvert. Rapamycin was given to achieve stable plasma levels between 4 and 8 ng/ml. AML volume was estimated using orthogonal measurements by MRI at baseline, 6, 12 and 24 months.
Ten out of 17 patients were success responders for the main outcome -58.8%, 95%CI: 32.9% to 81.6%-. After 6 months of therapy, the mean volume decrease was 55.18% (5.01 standard error (SE); p |
| doi_str_mv | 10.1186/1750-1172-7-87 |
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Twenty four-month prospective open-label, single arm, unicentre Phases II andIII study. The primary endpoint was to evaluate the effect of treatment on the reduction of at least 50% AML volume from baseline at 24 months. The secondary endpoints were: average tumour reduction, surgical complications, skin lesions and drug safety.The study population comprised 17 patients, aged >10 years who were diagnosed with TS and had ≥1 renal AML >2 cm of diameter and had a serum creatinine < 2mg/dl and urine protein/creatinine ratio < 22.6 mg/mmol. The trial was conducted at Fundació Puigvert. Rapamycin was given to achieve stable plasma levels between 4 and 8 ng/ml. AML volume was estimated using orthogonal measurements by MRI at baseline, 6, 12 and 24 months.
Ten out of 17 patients were success responders for the main outcome -58.8%, 95%CI: 32.9% to 81.6%-. After 6 months of therapy, the mean volume decrease was 55.18% (5.01 standard error (SE); p<0.001) and 66.38% (4.41 SE; p<0.001) at year 1. There was no significant decrease between year 1 and 2. According to RECIST criteria, all patients achieved a partial response at year 1 and all but two had already achieved this partial response after 6 months.The main analysis was performed according to the intention-to-treat principle analysis. Tumour volume was analyzed over time by means of mixed models for repeated measurement analysis. We used the baseline tumour volume as a covariate for the absolute change and percentage change from baseline data. The analysis was performed using SAS version 9.2 software, and the level of significance was established at 0.05 (two-sided).
This study show that mTOR inhibitors are a relatively safe, efficacious and less aggressive alternative than currently available options in the management of AML in TS.
EudraCT number: 2007-005978-30, ClinicalTrials.gov number: NCT0121712.</description><identifier>ISSN: 1750-1172</identifier><identifier>EISSN: 1750-1172</identifier><identifier>DOI: 10.1186/1750-1172-7-87</identifier><identifier>PMID: 23140536</identifier><language>eng</language><publisher>England: BioMed Central Ltd</publisher><subject>Adult ; Analysis ; Angiomyolipoma ; Angiomyolipoma - complications ; Angiomyolipoma - drug therapy ; Antibiotics, Antineoplastic - adverse effects ; Antibiotics, Antineoplastic - therapeutic use ; Dermatologic agents ; Dermatology ; Female ; Formulae, receipts, prescriptions ; Health aspects ; Humans ; Male ; Medical research ; Medicine, Experimental ; mTOR ; Prospective Studies ; Rapamycin ; Rare diseases ; Sirolimus - adverse effects ; Sirolimus - therapeutic use ; Skin ; Treatment ; Treatment Outcome ; Tuberous sclerosis ; Tuberous Sclerosis - complications</subject><ispartof>Orphanet journal of rare diseases, 2012-11, Vol.7 (1), p.87-87, Article 87</ispartof><rights>COPYRIGHT 2012 BioMed Central Ltd.</rights><rights>2012 López et al.; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.</rights><rights>Copyright ©2012 López et al.; licensee BioMed Central Ltd. 2012 López et al.; licensee BioMed Central Ltd.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-b643t-735460b9430ca70d98921ded1280c58b24a17eef93a1f95263c743f6cf80e2c43</citedby><cites>FETCH-LOGICAL-b643t-735460b9430ca70d98921ded1280c58b24a17eef93a1f95263c743f6cf80e2c43</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC3519505/pdf/$$EPDF$$P50$$Gpubmedcentral$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://www.proquest.com/docview/1223841626?pq-origsite=primo$$EHTML$$P50$$Gproquest$$Hfree_for_read</linktohtml><link.rule.ids>230,314,723,776,780,881,25731,27901,27902,36989,44566,53766,53768</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/23140536$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Cabrera-López, Cristina</creatorcontrib><creatorcontrib>Martí, Teresa</creatorcontrib><creatorcontrib>Catalá, Violeta</creatorcontrib><creatorcontrib>Torres, Ferran</creatorcontrib><creatorcontrib>Mateu, Silvia</creatorcontrib><creatorcontrib>Ballarín, Jose</creatorcontrib><creatorcontrib>Torra, Roser</creatorcontrib><title>Assessing the effectiveness of rapamycin on angiomyolipoma in tuberous sclerosis: a two years trial</title><title>Orphanet journal of rare diseases</title><addtitle>Orphanet J Rare Dis</addtitle><description>Tuberous sclerosis (TS) is a rare autosomal dominant systemic disease with an estimated prevalence of 1/6000. Renal angiomyolipoma (AML) is a benign tumour with high morbidity frequently present in TS. The aim of the study was to test the effect of rapamycin in reducing the volume of AML in TS.
Twenty four-month prospective open-label, single arm, unicentre Phases II andIII study. The primary endpoint was to evaluate the effect of treatment on the reduction of at least 50% AML volume from baseline at 24 months. The secondary endpoints were: average tumour reduction, surgical complications, skin lesions and drug safety.The study population comprised 17 patients, aged >10 years who were diagnosed with TS and had ≥1 renal AML >2 cm of diameter and had a serum creatinine < 2mg/dl and urine protein/creatinine ratio < 22.6 mg/mmol. The trial was conducted at Fundació Puigvert. Rapamycin was given to achieve stable plasma levels between 4 and 8 ng/ml. AML volume was estimated using orthogonal measurements by MRI at baseline, 6, 12 and 24 months.
Ten out of 17 patients were success responders for the main outcome -58.8%, 95%CI: 32.9% to 81.6%-. After 6 months of therapy, the mean volume decrease was 55.18% (5.01 standard error (SE); p<0.001) and 66.38% (4.41 SE; p<0.001) at year 1. There was no significant decrease between year 1 and 2. According to RECIST criteria, all patients achieved a partial response at year 1 and all but two had already achieved this partial response after 6 months.The main analysis was performed according to the intention-to-treat principle analysis. Tumour volume was analyzed over time by means of mixed models for repeated measurement analysis. We used the baseline tumour volume as a covariate for the absolute change and percentage change from baseline data. The analysis was performed using SAS version 9.2 software, and the level of significance was established at 0.05 (two-sided).
This study show that mTOR inhibitors are a relatively safe, efficacious and less aggressive alternative than currently available options in the management of AML in TS.
EudraCT number: 2007-005978-30, ClinicalTrials.gov number: NCT0121712.</description><subject>Adult</subject><subject>Analysis</subject><subject>Angiomyolipoma</subject><subject>Angiomyolipoma - complications</subject><subject>Angiomyolipoma - drug therapy</subject><subject>Antibiotics, Antineoplastic - adverse effects</subject><subject>Antibiotics, Antineoplastic - therapeutic use</subject><subject>Dermatologic agents</subject><subject>Dermatology</subject><subject>Female</subject><subject>Formulae, receipts, prescriptions</subject><subject>Health aspects</subject><subject>Humans</subject><subject>Male</subject><subject>Medical research</subject><subject>Medicine, Experimental</subject><subject>mTOR</subject><subject>Prospective Studies</subject><subject>Rapamycin</subject><subject>Rare diseases</subject><subject>Sirolimus - adverse effects</subject><subject>Sirolimus - therapeutic use</subject><subject>Skin</subject><subject>Treatment</subject><subject>Treatment Outcome</subject><subject>Tuberous sclerosis</subject><subject>Tuberous Sclerosis - complications</subject><issn>1750-1172</issn><issn>1750-1172</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2012</creationdate><recordtype>article</recordtype><sourceid>PIMPY</sourceid><sourceid>DOA</sourceid><recordid>eNptkk2P0zAQhiMEYpfClSOyxIlDFju2Y4cDUrXio9JKSHycrYljZ101cbGdhf57HLpUG1H54NE7M49mXrsoXhJ8RYis3xLBcUmIqEpRSvGouDwJjx_EF8WzGLcYM06xfFpcVJQwzGl9Weh1jCZGN_Yo3RpkrDU6uTszZhF5iwLsYThoNyI_Ihh754eD37m9HwBlMU2tCX6KKOpdDqKL7xCg9Mujg4EQUQoOds-LJxZ20by4v1fFj48fvl9_Lm--fNpcr2_KtmY0lYJyVuO2YRRrELhrZFORznSkklhz2VYMiDDGNhSIbXhVUy0YtbW2EptKM7oqNkdu52Gr9sENEA7Kg1N_BR96BSG5PKniouEwO2M4ZoS2bUdBAKOSd23XEJpZ74-s_dQOptNmTAF2C-gyM7pb1fs7RTlpePZ2VayPgDZbdh6wzGg_qPnB1DyWEkqKzHh9P0TwPycTk9r6KYzZQ0WqikpG6uzCqaqHvJkbrc88Pbio1ZpTRishyDzP1ZmqfDozOO1HY13WFw1vFg25JpnfqYcpRrX59vUsXOc_EIOxpz3JvI-s_9_s1UN7T-X_Pib9A8lf4a0</recordid><startdate>20121111</startdate><enddate>20121111</enddate><creator>Cabrera-López, Cristina</creator><creator>Martí, Teresa</creator><creator>Catalá, Violeta</creator><creator>Torres, Ferran</creator><creator>Mateu, Silvia</creator><creator>Ballarín, Jose</creator><creator>Torra, Roser</creator><general>BioMed Central Ltd</general><general>BioMed Central</general><general>BMC</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>ISR</scope><scope>3V.</scope><scope>7T5</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AN0</scope><scope>AZQEC</scope><scope>BENPR</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>H94</scope><scope>K9.</scope><scope>M0S</scope><scope>M1P</scope><scope>PIMPY</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>5PM</scope><scope>DOA</scope></search><sort><creationdate>20121111</creationdate><title>Assessing the effectiveness of rapamycin on angiomyolipoma in tuberous sclerosis: a two years trial</title><author>Cabrera-López, Cristina ; Martí, Teresa ; Catalá, Violeta ; Torres, Ferran ; Mateu, Silvia ; Ballarín, Jose ; Torra, Roser</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-b643t-735460b9430ca70d98921ded1280c58b24a17eef93a1f95263c743f6cf80e2c43</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2012</creationdate><topic>Adult</topic><topic>Analysis</topic><topic>Angiomyolipoma</topic><topic>Angiomyolipoma - complications</topic><topic>Angiomyolipoma - drug therapy</topic><topic>Antibiotics, Antineoplastic - adverse effects</topic><topic>Antibiotics, Antineoplastic - therapeutic use</topic><topic>Dermatologic agents</topic><topic>Dermatology</topic><topic>Female</topic><topic>Formulae, receipts, prescriptions</topic><topic>Health aspects</topic><topic>Humans</topic><topic>Male</topic><topic>Medical research</topic><topic>Medicine, Experimental</topic><topic>mTOR</topic><topic>Prospective Studies</topic><topic>Rapamycin</topic><topic>Rare diseases</topic><topic>Sirolimus - adverse effects</topic><topic>Sirolimus - therapeutic use</topic><topic>Skin</topic><topic>Treatment</topic><topic>Treatment Outcome</topic><topic>Tuberous sclerosis</topic><topic>Tuberous Sclerosis - complications</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Cabrera-López, Cristina</creatorcontrib><creatorcontrib>Martí, Teresa</creatorcontrib><creatorcontrib>Catalá, Violeta</creatorcontrib><creatorcontrib>Torres, Ferran</creatorcontrib><creatorcontrib>Mateu, Silvia</creatorcontrib><creatorcontrib>Ballarín, Jose</creatorcontrib><creatorcontrib>Torra, Roser</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Science in Context</collection><collection>ProQuest Central (Corporate)</collection><collection>Immunology Abstracts</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Medical Database (Alumni Edition)</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>ProQuest Central (Alumni)</collection><collection>ProQuest Central</collection><collection>British Nursing Database</collection><collection>ProQuest Central Essentials</collection><collection>ProQuest Central</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Medical Database</collection><collection>Publicly Available Content Database</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>PubMed Central (Full Participant titles)</collection><collection>DOAJ Directory of Open Access Journals</collection><jtitle>Orphanet journal of rare diseases</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Cabrera-López, Cristina</au><au>Martí, Teresa</au><au>Catalá, Violeta</au><au>Torres, Ferran</au><au>Mateu, Silvia</au><au>Ballarín, Jose</au><au>Torra, Roser</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Assessing the effectiveness of rapamycin on angiomyolipoma in tuberous sclerosis: a two years trial</atitle><jtitle>Orphanet journal of rare diseases</jtitle><addtitle>Orphanet J Rare Dis</addtitle><date>2012-11-11</date><risdate>2012</risdate><volume>7</volume><issue>1</issue><spage>87</spage><epage>87</epage><pages>87-87</pages><artnum>87</artnum><issn>1750-1172</issn><eissn>1750-1172</eissn><abstract>Tuberous sclerosis (TS) is a rare autosomal dominant systemic disease with an estimated prevalence of 1/6000. Renal angiomyolipoma (AML) is a benign tumour with high morbidity frequently present in TS. The aim of the study was to test the effect of rapamycin in reducing the volume of AML in TS.
Twenty four-month prospective open-label, single arm, unicentre Phases II andIII study. The primary endpoint was to evaluate the effect of treatment on the reduction of at least 50% AML volume from baseline at 24 months. The secondary endpoints were: average tumour reduction, surgical complications, skin lesions and drug safety.The study population comprised 17 patients, aged >10 years who were diagnosed with TS and had ≥1 renal AML >2 cm of diameter and had a serum creatinine < 2mg/dl and urine protein/creatinine ratio < 22.6 mg/mmol. The trial was conducted at Fundació Puigvert. Rapamycin was given to achieve stable plasma levels between 4 and 8 ng/ml. AML volume was estimated using orthogonal measurements by MRI at baseline, 6, 12 and 24 months.
Ten out of 17 patients were success responders for the main outcome -58.8%, 95%CI: 32.9% to 81.6%-. After 6 months of therapy, the mean volume decrease was 55.18% (5.01 standard error (SE); p<0.001) and 66.38% (4.41 SE; p<0.001) at year 1. There was no significant decrease between year 1 and 2. According to RECIST criteria, all patients achieved a partial response at year 1 and all but two had already achieved this partial response after 6 months.The main analysis was performed according to the intention-to-treat principle analysis. Tumour volume was analyzed over time by means of mixed models for repeated measurement analysis. We used the baseline tumour volume as a covariate for the absolute change and percentage change from baseline data. The analysis was performed using SAS version 9.2 software, and the level of significance was established at 0.05 (two-sided).
This study show that mTOR inhibitors are a relatively safe, efficacious and less aggressive alternative than currently available options in the management of AML in TS.
EudraCT number: 2007-005978-30, ClinicalTrials.gov number: NCT0121712.</abstract><cop>England</cop><pub>BioMed Central Ltd</pub><pmid>23140536</pmid><doi>10.1186/1750-1172-7-87</doi><tpages>1</tpages><oa>free_for_read</oa></addata></record> |
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| subjects | Adult Analysis Angiomyolipoma Angiomyolipoma - complications Angiomyolipoma - drug therapy Antibiotics, Antineoplastic - adverse effects Antibiotics, Antineoplastic - therapeutic use Dermatologic agents Dermatology Female Formulae, receipts, prescriptions Health aspects Humans Male Medical research Medicine, Experimental mTOR Prospective Studies Rapamycin Rare diseases Sirolimus - adverse effects Sirolimus - therapeutic use Skin Treatment Treatment Outcome Tuberous sclerosis Tuberous Sclerosis - complications |
| title | Assessing the effectiveness of rapamycin on angiomyolipoma in tuberous sclerosis: a two years trial |
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