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Exon-Skipping in Duchenne Muscular Dystrophy

05 December 24 Articles ، Eric P. Hoffman ، Paula R. Clemens ، Shin’ichi Takeda Micro-dystrophin gene therapy ، Duchenne muscular dystrophy exon skipping ، Antisense oligonucleotides (AONs) for DMD ، Eteplirsen and viltolarsen therapy ، Becker muscular dystrophy phenotype conversion ، Dystrophin restoration therapies ، Stop codon read-through drugs for DMD ، Future treatments for muscular dystrophy
Exon-Skipping in Duchenne Muscular Dystrophy
Exon-skipping therapy aims to restore dystrophin production in Duchenne muscular dystrophy (DMD) by using antisense oligonucleotides (AONs) to bypass mutations. Eteplirsen and viltolarsen target exon 51, producing a truncated but functional dystrophin protein. While promising, challenges remain, including low dystrophin levels and variable patient responses. Gene therapy and stop codon read-through drugs offer alternative strategies. Early intervention and combination therapies may improve outcomes for DMD patients.
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Adrenal Suppression From Vamorolone and Prednisone in Duchenne Muscular Dystrophy: Results From the Phase 2b Clinical Trial

03 September 24 Articles ، Utkarsh J. Dang ، Eric P. Hoffman ، Michela Guglieri ، Leanne Ward ، Paula R. Clemens Adrenal suppression in Duchenne dystrophy ، Vamorolone vs prednisone in DMD treatment ، Cortisol thresholds in adrenal function ، Steroid therapy side effects in DMD ، Hydrocortisone for stress dosing in DMD ، ACTH-stimulated cortisol in muscular dystrophy ، Phase 2b clinical trial on vamorolone
Adrenal Suppression From Vamorolone and Prednisone in Duchenne Muscular Dystrophy: Results From the Phase 2b Clinical Trial
This study examines adrenal suppression in Duchenne muscular dystrophy patients treated with vamorolone and prednisone, emphasizing cortisol thresholds and management strategies.
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Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to

16 September 18 Articles ، Kanneboyina Nagaraju ، Eric P. Hoffman ، Jack H Vandermeulen LGMD2B ، Vamorolone ، LGMD2B therapy ، Dysferlin deficiency treatment ، Muscle membrane stabilization ، Vamorolone vs Prednisolone ، Steroid treatment for muscular dystrophy ، Dysferlin protein repair ، Clinical trials for LGMD2B ، Muscular Dystrophy
Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to
This study explores the potential of membrane stabilization therapy for LGMD2B using vamorolone, showing it outperforms prednisolone in muscle repair and strength restoration.
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