Journal: Chemistry & Biology

Article Title: The Iterative Gramicidin S Thioesterase Catalyzes Peptide Ligation and Cyclization

doi: 10.1016/j.chembiol.2006.10.011

Figure Lengend Snippet: Dimerization and Cyclization of Pentapeptidyl- and Decapeptidyl-Thioesters Mediated by the GrsB PCP-TE (A) Incubation of GS10 (300 μM) with PCP-TE (10 μM) for 60 min. (B) Incubation of GS5 SNAC (300 μM) with PCP-TE (10 μM) for 60 min. (C) Incubation of GS5 SPh (300 μM) with PCP-TE (10 μM) for 60 min. The blue HPLC trace corresponds to the assay with enzyme, and the red trace shows incubation of the substrate in the absence of the enzyme. Analysis was performed on a C 18 Nucleodur column (Macherey and Nagel; 250/2; pore diameter, 100 Å; particle size, 3 μM) with the following gradient: 0–40 min, 5%–60% MeCN/0.1% TFA into H 2 O/0.1% TFA, 40–45 min, 60%–95% MeCN/0.1% TFA into H 2 O/0.1% TFA, 0.3 ml/min, 45°C.

Article Snippet: Analysis was performed on a C 18 Nucleodur column (Macherey and Nagel; 250/2; pore diameter, 100 Å; particle size, 3 μM) with the following gradient: 0–40 min, 5%–60% MeCN/0.1% TFA into H 2 O/0.1% TFA, 40–45 min, 60%–95% MeCN/0.1% TFA into H 2 O/0.1% TFA, 0.3 ml/min, 45°C.

Techniques: Incubation

Journal: Orphanet Journal of Rare Diseases

Article Title: Newborn screening for Morquio disease and other lysosomal storage diseases: results from the 8-plex assay for 70,000 newborns

doi: 10.1186/s13023-020-1322-z

Figure Lengend Snippet: Genotypes and biomarkers level of patients identified in this study except for MPS 4A

Article Snippet: Sanofi Genzyme funds partially the biomarkers analysis.

Techniques:

Journal: The Cochrane Database of Systematic Reviews

Article Title: Interventions for idiopathic steroid‐resistant nephrotic syndrome in children

doi: 10.1002/14651858.CD003594.pub6

Figure Lengend Snippet: Cyclosporin versus placebo or no treatment for idiopathic steroid‐resistant nephrotic syndrome in children

Article Snippet: A secondary outcome defined by the authors of "FSGS partial remission end point (FPRE) (UP/C:≤ 1.5 g/g and > 40% reduction in proteinuria" and not pre‐specified in the protocol was included in meta‐analyses Other bias High risk Trial organised and supported by Retrophin Inc. (San Diego, CA) DUET 2017 Methods Study design: parallel RCT Time frame: 1990 to 1991 Follow‐up period: 12 months Participants Setting: tertiary centre Country: India SRNS, initial (5) and delayed (8) steroid resistance with MCD Number (IV/oral): 7/6 Age range (years): IV group (3 to 16); oral group (9 to 14.5) Sex (M/F): IV group (6/1); oral group (5/1) Exclusion criteria: not reported Interventions IV CPA group IV CPA: 500 mg/m 2 /mo for 6 months Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Oral CPA group Oral CPA: 2.5 mg/kg/d for 8 weeks Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Co‐interventions Not reported Outcomes Remission: proteinuria < 4 mg/m 2 /h and albumin > 35 g/L at 6 months Adverse events Notes Exclusions post randomisation but pre‐intervention: none reported Stop or end points/s: not reported Additional data requested from authors: none Risk of bias Bias Authors' judgement Support for judgement Random sequence generation (selection bias) Unclear risk No information provided Allocation concealment (selection bias) Unclear risk No information provided Blinding of participants and personnel (performance bias) All outcomes High risk No blinding of participants/investigators; lack of blinding could influence management Blinding of outcome assessment (detection bias) All outcomes Low risk Primary outcome was laboratory based and unlikely to be influenced by lack of blinding Incomplete outcome data (attrition bias) All outcomes High risk Loss to follow‐up: 15%; 2 from control group lost to follow‐up and excluded from analysis Selective reporting (reporting bias) Low risk Outcome (complete remission, non‐remission, adverse effects) reported Other bias Unclear risk Funding source not reported Elhence 1994 Methods Study design: parallel RCT (phase 1 study) Time frame: not reported Follow‐up period: 16 weeks Participants Setting: multicentre Country: USA Adults and children aged 2 to 41 years with biopsy‐confirmed primary FSGS and initial steroid resistance; steroid resistance (UP/C > 1.0 g/g after 4 weeks of steroid therapy), persistent proteinuria (UP/C > 1.0 g/g) and eGFR > 40 mL/min/1.73 m 2 ; patients were admitted who failed treatment in the FSGS‐CT Study 2011 or were ineligible for FSGS‐CT Study because of previous use of study interventions; patients off all immunosuppressive agents for at least 4 weeks.

Techniques: Comparison, Infection

Journal: The Cochrane Database of Systematic Reviews

Article Title: Interventions for idiopathic steroid‐resistant nephrotic syndrome in children

doi: 10.1002/14651858.CD003594.pub6

Figure Lengend Snippet: Oral cyclophosphamide versus prednisone or placebo for idiopathic steroid‐resistant nephrotic syndrome in children

Article Snippet: A secondary outcome defined by the authors of "FSGS partial remission end point (FPRE) (UP/C:≤ 1.5 g/g and > 40% reduction in proteinuria" and not pre‐specified in the protocol was included in meta‐analyses Other bias High risk Trial organised and supported by Retrophin Inc. (San Diego, CA) DUET 2017 Methods Study design: parallel RCT Time frame: 1990 to 1991 Follow‐up period: 12 months Participants Setting: tertiary centre Country: India SRNS, initial (5) and delayed (8) steroid resistance with MCD Number (IV/oral): 7/6 Age range (years): IV group (3 to 16); oral group (9 to 14.5) Sex (M/F): IV group (6/1); oral group (5/1) Exclusion criteria: not reported Interventions IV CPA group IV CPA: 500 mg/m 2 /mo for 6 months Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Oral CPA group Oral CPA: 2.5 mg/kg/d for 8 weeks Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Co‐interventions Not reported Outcomes Remission: proteinuria < 4 mg/m 2 /h and albumin > 35 g/L at 6 months Adverse events Notes Exclusions post randomisation but pre‐intervention: none reported Stop or end points/s: not reported Additional data requested from authors: none Risk of bias Bias Authors' judgement Support for judgement Random sequence generation (selection bias) Unclear risk No information provided Allocation concealment (selection bias) Unclear risk No information provided Blinding of participants and personnel (performance bias) All outcomes High risk No blinding of participants/investigators; lack of blinding could influence management Blinding of outcome assessment (detection bias) All outcomes Low risk Primary outcome was laboratory based and unlikely to be influenced by lack of blinding Incomplete outcome data (attrition bias) All outcomes High risk Loss to follow‐up: 15%; 2 from control group lost to follow‐up and excluded from analysis Selective reporting (reporting bias) Low risk Outcome (complete remission, non‐remission, adverse effects) reported Other bias Unclear risk Funding source not reported Elhence 1994 Methods Study design: parallel RCT (phase 1 study) Time frame: not reported Follow‐up period: 16 weeks Participants Setting: multicentre Country: USA Adults and children aged 2 to 41 years with biopsy‐confirmed primary FSGS and initial steroid resistance; steroid resistance (UP/C > 1.0 g/g after 4 weeks of steroid therapy), persistent proteinuria (UP/C > 1.0 g/g) and eGFR > 40 mL/min/1.73 m 2 ; patients were admitted who failed treatment in the FSGS‐CT Study 2011 or were ineligible for FSGS‐CT Study because of previous use of study interventions; patients off all immunosuppressive agents for at least 4 weeks.

Techniques: Comparison

Journal: The Cochrane Database of Systematic Reviews

Article Title: Interventions for idiopathic steroid‐resistant nephrotic syndrome in children

doi: 10.1002/14651858.CD003594.pub6

Figure Lengend Snippet:

Article Snippet: A secondary outcome defined by the authors of "FSGS partial remission end point (FPRE) (UP/C:≤ 1.5 g/g and > 40% reduction in proteinuria" and not pre‐specified in the protocol was included in meta‐analyses Other bias High risk Trial organised and supported by Retrophin Inc. (San Diego, CA) DUET 2017 Methods Study design: parallel RCT Time frame: 1990 to 1991 Follow‐up period: 12 months Participants Setting: tertiary centre Country: India SRNS, initial (5) and delayed (8) steroid resistance with MCD Number (IV/oral): 7/6 Age range (years): IV group (3 to 16); oral group (9 to 14.5) Sex (M/F): IV group (6/1); oral group (5/1) Exclusion criteria: not reported Interventions IV CPA group IV CPA: 500 mg/m 2 /mo for 6 months Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Oral CPA group Oral CPA: 2.5 mg/kg/d for 8 weeks Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Co‐interventions Not reported Outcomes Remission: proteinuria < 4 mg/m 2 /h and albumin > 35 g/L at 6 months Adverse events Notes Exclusions post randomisation but pre‐intervention: none reported Stop or end points/s: not reported Additional data requested from authors: none Risk of bias Bias Authors' judgement Support for judgement Random sequence generation (selection bias) Unclear risk No information provided Allocation concealment (selection bias) Unclear risk No information provided Blinding of participants and personnel (performance bias) All outcomes High risk No blinding of participants/investigators; lack of blinding could influence management Blinding of outcome assessment (detection bias) All outcomes Low risk Primary outcome was laboratory based and unlikely to be influenced by lack of blinding Incomplete outcome data (attrition bias) All outcomes High risk Loss to follow‐up: 15%; 2 from control group lost to follow‐up and excluded from analysis Selective reporting (reporting bias) Low risk Outcome (complete remission, non‐remission, adverse effects) reported Other bias Unclear risk Funding source not reported Elhence 1994 Methods Study design: parallel RCT (phase 1 study) Time frame: not reported Follow‐up period: 16 weeks Participants Setting: multicentre Country: USA Adults and children aged 2 to 41 years with biopsy‐confirmed primary FSGS and initial steroid resistance; steroid resistance (UP/C > 1.0 g/g after 4 weeks of steroid therapy), persistent proteinuria (UP/C > 1.0 g/g) and eGFR > 40 mL/min/1.73 m 2 ; patients were admitted who failed treatment in the FSGS‐CT Study 2011 or were ineligible for FSGS‐CT Study because of previous use of study interventions; patients off all immunosuppressive agents for at least 4 weeks.

Techniques:

Journal: The Cochrane Database of Systematic Reviews

Article Title: Interventions for idiopathic steroid‐resistant nephrotic syndrome in children

doi: 10.1002/14651858.CD003594.pub6

Figure Lengend Snippet: Cyclosporin (CSA) versus placebo/no treatment

Article Snippet: A secondary outcome defined by the authors of "FSGS partial remission end point (FPRE) (UP/C:≤ 1.5 g/g and > 40% reduction in proteinuria" and not pre‐specified in the protocol was included in meta‐analyses Other bias High risk Trial organised and supported by Retrophin Inc. (San Diego, CA) DUET 2017 Methods Study design: parallel RCT Time frame: 1990 to 1991 Follow‐up period: 12 months Participants Setting: tertiary centre Country: India SRNS, initial (5) and delayed (8) steroid resistance with MCD Number (IV/oral): 7/6 Age range (years): IV group (3 to 16); oral group (9 to 14.5) Sex (M/F): IV group (6/1); oral group (5/1) Exclusion criteria: not reported Interventions IV CPA group IV CPA: 500 mg/m 2 /mo for 6 months Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Oral CPA group Oral CPA: 2.5 mg/kg/d for 8 weeks Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Co‐interventions Not reported Outcomes Remission: proteinuria < 4 mg/m 2 /h and albumin > 35 g/L at 6 months Adverse events Notes Exclusions post randomisation but pre‐intervention: none reported Stop or end points/s: not reported Additional data requested from authors: none Risk of bias Bias Authors' judgement Support for judgement Random sequence generation (selection bias) Unclear risk No information provided Allocation concealment (selection bias) Unclear risk No information provided Blinding of participants and personnel (performance bias) All outcomes High risk No blinding of participants/investigators; lack of blinding could influence management Blinding of outcome assessment (detection bias) All outcomes Low risk Primary outcome was laboratory based and unlikely to be influenced by lack of blinding Incomplete outcome data (attrition bias) All outcomes High risk Loss to follow‐up: 15%; 2 from control group lost to follow‐up and excluded from analysis Selective reporting (reporting bias) Low risk Outcome (complete remission, non‐remission, adverse effects) reported Other bias Unclear risk Funding source not reported Elhence 1994 Methods Study design: parallel RCT (phase 1 study) Time frame: not reported Follow‐up period: 16 weeks Participants Setting: multicentre Country: USA Adults and children aged 2 to 41 years with biopsy‐confirmed primary FSGS and initial steroid resistance; steroid resistance (UP/C > 1.0 g/g after 4 weeks of steroid therapy), persistent proteinuria (UP/C > 1.0 g/g) and eGFR > 40 mL/min/1.73 m 2 ; patients were admitted who failed treatment in the FSGS‐CT Study 2011 or were ineligible for FSGS‐CT Study because of previous use of study interventions; patients off all immunosuppressive agents for at least 4 weeks.

Techniques: Infection

Journal: The Cochrane Database of Systematic Reviews

Article Title: Interventions for idiopathic steroid‐resistant nephrotic syndrome in children

doi: 10.1002/14651858.CD003594.pub6

Figure Lengend Snippet: Oral cyclophosphamide (CPA) versus prednisone/placebo

Article Snippet: A secondary outcome defined by the authors of "FSGS partial remission end point (FPRE) (UP/C:≤ 1.5 g/g and > 40% reduction in proteinuria" and not pre‐specified in the protocol was included in meta‐analyses Other bias High risk Trial organised and supported by Retrophin Inc. (San Diego, CA) DUET 2017 Methods Study design: parallel RCT Time frame: 1990 to 1991 Follow‐up period: 12 months Participants Setting: tertiary centre Country: India SRNS, initial (5) and delayed (8) steroid resistance with MCD Number (IV/oral): 7/6 Age range (years): IV group (3 to 16); oral group (9 to 14.5) Sex (M/F): IV group (6/1); oral group (5/1) Exclusion criteria: not reported Interventions IV CPA group IV CPA: 500 mg/m 2 /mo for 6 months Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Oral CPA group Oral CPA: 2.5 mg/kg/d for 8 weeks Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Co‐interventions Not reported Outcomes Remission: proteinuria < 4 mg/m 2 /h and albumin > 35 g/L at 6 months Adverse events Notes Exclusions post randomisation but pre‐intervention: none reported Stop or end points/s: not reported Additional data requested from authors: none Risk of bias Bias Authors' judgement Support for judgement Random sequence generation (selection bias) Unclear risk No information provided Allocation concealment (selection bias) Unclear risk No information provided Blinding of participants and personnel (performance bias) All outcomes High risk No blinding of participants/investigators; lack of blinding could influence management Blinding of outcome assessment (detection bias) All outcomes Low risk Primary outcome was laboratory based and unlikely to be influenced by lack of blinding Incomplete outcome data (attrition bias) All outcomes High risk Loss to follow‐up: 15%; 2 from control group lost to follow‐up and excluded from analysis Selective reporting (reporting bias) Low risk Outcome (complete remission, non‐remission, adverse effects) reported Other bias Unclear risk Funding source not reported Elhence 1994 Methods Study design: parallel RCT (phase 1 study) Time frame: not reported Follow‐up period: 16 weeks Participants Setting: multicentre Country: USA Adults and children aged 2 to 41 years with biopsy‐confirmed primary FSGS and initial steroid resistance; steroid resistance (UP/C > 1.0 g/g after 4 weeks of steroid therapy), persistent proteinuria (UP/C > 1.0 g/g) and eGFR > 40 mL/min/1.73 m 2 ; patients were admitted who failed treatment in the FSGS‐CT Study 2011 or were ineligible for FSGS‐CT Study because of previous use of study interventions; patients off all immunosuppressive agents for at least 4 weeks.

Techniques:

Journal: The Cochrane Database of Systematic Reviews

Article Title: Interventions for idiopathic steroid‐resistant nephrotic syndrome in children

doi: 10.1002/14651858.CD003594.pub6

Figure Lengend Snippet: IV versus oral cyclophosphamide (CPA) plus IV dexamethasone (DEXA)

Article Snippet: A secondary outcome defined by the authors of "FSGS partial remission end point (FPRE) (UP/C:≤ 1.5 g/g and > 40% reduction in proteinuria" and not pre‐specified in the protocol was included in meta‐analyses Other bias High risk Trial organised and supported by Retrophin Inc. (San Diego, CA) DUET 2017 Methods Study design: parallel RCT Time frame: 1990 to 1991 Follow‐up period: 12 months Participants Setting: tertiary centre Country: India SRNS, initial (5) and delayed (8) steroid resistance with MCD Number (IV/oral): 7/6 Age range (years): IV group (3 to 16); oral group (9 to 14.5) Sex (M/F): IV group (6/1); oral group (5/1) Exclusion criteria: not reported Interventions IV CPA group IV CPA: 500 mg/m 2 /mo for 6 months Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Oral CPA group Oral CPA: 2.5 mg/kg/d for 8 weeks Prednisone: 60 mg/m 2 /d for 4 weeks; 40 mg/m 2 alternate days for 4 weeks and taper Co‐interventions Not reported Outcomes Remission: proteinuria < 4 mg/m 2 /h and albumin > 35 g/L at 6 months Adverse events Notes Exclusions post randomisation but pre‐intervention: none reported Stop or end points/s: not reported Additional data requested from authors: none Risk of bias Bias Authors' judgement Support for judgement Random sequence generation (selection bias) Unclear risk No information provided Allocation concealment (selection bias) Unclear risk No information provided Blinding of participants and personnel (performance bias) All outcomes High risk No blinding of participants/investigators; lack of blinding could influence management Blinding of outcome assessment (detection bias) All outcomes Low risk Primary outcome was laboratory based and unlikely to be influenced by lack of blinding Incomplete outcome data (attrition bias) All outcomes High risk Loss to follow‐up: 15%; 2 from control group lost to follow‐up and excluded from analysis Selective reporting (reporting bias) Low risk Outcome (complete remission, non‐remission, adverse effects) reported Other bias Unclear risk Funding source not reported Elhence 1994 Methods Study design: parallel RCT (phase 1 study) Time frame: not reported Follow‐up period: 16 weeks Participants Setting: multicentre Country: USA Adults and children aged 2 to 41 years with biopsy‐confirmed primary FSGS and initial steroid resistance; steroid resistance (UP/C > 1.0 g/g after 4 weeks of steroid therapy), persistent proteinuria (UP/C > 1.0 g/g) and eGFR > 40 mL/min/1.73 m 2 ; patients were admitted who failed treatment in the FSGS‐CT Study 2011 or were ineligible for FSGS‐CT Study because of previous use of study interventions; patients off all immunosuppressive agents for at least 4 weeks.

Techniques: