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Longitudinal Effects of Glucose-Lowering Medications on β-Cell Responses and Insulin Sensitivity in Type 2 Diabetes: The GRADE Randomized Clinical Trial.
Rasouli N, Younes N, Ghosh A, Albu J, Cohen RM, DeFronzo RA, Diaz E, Sayyed Kassem L, Luchsinger JA, McGill JB, Sivitz WI, Tamborlane WV, Utzschneider KM, Kahn SE; GRADE Research Group. Rasouli N, et al. Diabetes Care. 2024 Apr 1;47(4):580-588. doi: 10.2337/dc23-1070. Diabetes Care. 2024. PMID: 38211595 Clinical Trial.
OBJECTIVE: To compare the long-term effects of glucose-lowering medications (insulin glargine U-100, glimepiride, liraglutide, and sitagliptin) when added to metformin on insulin sensitivity and beta-cell function. ...After adjustment for HOMA2-%S to obtain an estimate of …
OBJECTIVE: To compare the long-term effects of glucose-lowering medications (insulin glargine U-100, glimepiride, liraglutide, and si …
Impact of Insulin Sensitivity and β-Cell Function Over Time on Glycemic Outcomes in the Glycemia Reduction Approaches in Diabetes: A Comparative Effectiveness Study (GRADE): Differential Treatment Effects of Dual Therapy.
Utzschneider KM, Younes N, Butera NM, Balasubramanyam A, Bergenstal RM, Barzilay J, DeSouza C, DeFronzo RA, Elasy T, Krakoff J, Kahn SE, Rasouli N, Valencia WM, Sivitz WI; GRADE Research Group. Utzschneider KM, et al. Diabetes Care. 2024 Apr 1;47(4):571-579. doi: 10.2337/dc23-1059. Diabetes Care. 2024. PMID: 38190619 Clinical Trial.
CONCLUSIONS: Insulin sensitivity and beta-cell function affected treatment outcomes irrespective of drug assignment, with greater impact in the sitagliptin group on initial (short-term) HbA1c response in comparison with the glimepiride and liraglutide groups....
CONCLUSIONS: Insulin sensitivity and beta-cell function affected treatment outcomes irrespective of drug assignment, with greater impact in …
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results.
Mendell JR, Pozsgai ER, Lewis S, Griffin DA, Lowes LP, Alfano LN, Lehman KJ, Church K, Reash NF, Iammarino MA, Sabo B, Potter R, Neuhaus S, Li X, Stevenson H, Rodino-Klapac LR. Mendell JR, et al. Among authors: alfano ln. Nat Med. 2024 Jan;30(1):199-206. doi: 10.1038/s41591-023-02730-9. Epub 2024 Jan 4. Nat Med. 2024. PMID: 38177855 Free PMC article. Clinical Trial.
The 2-year safety and efficacy of bidridistrogene xeboparvovec support clinical development advancement. Further studies are necessary to confirm the long-term safety and efficacy of this gene therapy. ClinicalTrials.gov registration: NCT03652259 ....
The 2-year safety and efficacy of bidridistrogene xeboparvovec support clinical development advancement. Further studies are necessary to co …
Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio.
Waldrop MA, Chagat S, Storey M, Meyer A, Iammarino M, Reash N, Alfano L, Lowes L, Noritz G, Prochoroff A, Rossman I, Ginsberg M, Mosher K, Broomall E, Bass N, Gushue C, Kotha K, Paul G, Shell R, Tsao CY, Mendell JR, Connolly AM. Waldrop MA, et al. Among authors: alfano l. Neuromuscul Disord. 2024 Jan;34:41-48. doi: 10.1016/j.nmd.2023.11.010. Epub 2023 Dec 2. Neuromuscul Disord. 2024. PMID: 38142474
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Mendell JR, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Lewis S, Church K, Shell R, Potter RA, Griffin DA, Hogan M, Wang S, Mason S, Darton E, Rodino-Klapac LR. Mendell JR, et al. Among authors: alfano ln. Muscle Nerve. 2024 Jan;69(1):93-98. doi: 10.1002/mus.27955. Epub 2023 Aug 14. Muscle Nerve. 2024. PMID: 37577753 Clinical Trial.
INTRODUCTION/AIMS: Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. Long-term delandistrogene moxeparvo …
INTRODUCTION/AIMS: Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 …
Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
Mendell JR, Al-Zaidy SA, Lehman KJ, McColly M, Lowes LP, Alfano LN, Reash NF, Iammarino MA, Church KR, Kleyn A, Meriggioli MN, Shell R. Mendell JR, et al. Among authors: alfano ln. JAMA Neurol. 2021 Jul 1;78(7):834-841. doi: 10.1001/jamaneurol.2021.1272. JAMA Neurol. 2021. PMID: 33999158 Free PMC article. Clinical Trial.
IMPORTANCE: This ongoing study assesses long-term safety and durability of response in infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene abeparvovec gene replacement therapy. ...CONCLUSIONS AND RELEVANCE: The findings of this ongoing clinical …
IMPORTANCE: This ongoing study assesses long-term safety and durability of response in infants with spinal muscular atrophy (SMA) typ …
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale.
Jacobs MB, James MK, Lowes LP, Alfano LN, Eagle M, Muni Lofra R, Moore U, Feng J, Rufibach LE, Rose K, Duong T, Bello L, Pedrosa-Hernández I, Holsten S, Sakamoto C, Canal A, Sanchez-Aguilera Práxedes N, Thiele S, Siener C, Vandevelde B, DeWolf B, Maron E, Guglieri M, Hogrel JY, Blamire AM, Carlier PG, Spuler S, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Pestronk A, Walter MC, Paradas C, Stojkovic T, Mori-Yoshimura M, Bravver E, Díaz-Manera J, Pegoraro E, Mendell JR; Jain COS Consortium; Mayhew AG, Straub V. Jacobs MB, et al. Among authors: alfano ln. Ann Neurol. 2021 May;89(5):967-978. doi: 10.1002/ana.26044. Epub 2021 Feb 26. Ann Neurol. 2021. PMID: 33576057 Free article.
Longitudinal analysis showed it may be possible to identify patient factors associated with greater functional decline both across the disease course and in the short-term for clinical trial preparation. Through further work and validation in this cohort, we anticipate tha …
Longitudinal analysis showed it may be possible to identify patient factors associated with greater functional decline both across the disea …
Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls.
Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN; Eteplirsen Study Group. Mendell JR, et al. Among authors: alfano ln. J Neuromuscul Dis. 2021;8(4):469-479. doi: 10.3233/JND-200548. J Neuromuscul Dis. 2021. PMID: 33523015 Free PMC article.
OBJECTIVE: Compare long-term ambulatory function in eteplirsen-treated patients versus controls. ...CONCLUSION: In this retrospective matched control study, eteplirsen treatment resulted in attenuation of ambulatory decline over a 4-year observation period, supporting long …
OBJECTIVE: Compare long-term ambulatory function in eteplirsen-treated patients versus controls. ...CONCLUSION: In this retrospective …
Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy.
Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Alfano LN, et al. Medicine (Baltimore). 2019 Jun;98(26):e15858. doi: 10.1097/MD.0000000000015858. Medicine (Baltimore). 2019. PMID: 31261494 Free PMC article. Clinical Trial.
All 12 patients continued onto open-label extension study 202 and received long-term treatment with eteplirsen. We compared cardiac, pulmonary, and upper limb function and dystrophin production in the nonambulatory twin patients versus the 10 ambulatory patients through 24 …
All 12 patients continued onto open-label extension study 202 and received long-term treatment with eteplirsen. We compared cardiac, …
Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy.
Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Ke Q, et al. Among authors: alfano ln. World J Pediatr. 2019 Jun;15(3):219-225. doi: 10.1007/s12519-019-00242-6. Epub 2019 Mar 23. World J Pediatr. 2019. PMID: 30904991 Review.
CONCLUSIONS: Approved treatments for DMD and SMA may change the natural history of these diseases. Long-term studies of these treatments are underway. To avoid the known diagnostic delay associated with these disorders and provide optimal effectiveness of these treatments, …
CONCLUSIONS: Approved treatments for DMD and SMA may change the natural history of these diseases. Long-term studies of these treatme …
13 results