Gene Therapy for Cardiomyopathy Associated With Friedreich’s Ataxia Post author: Post published:March 25, 2026 Post category: Continue ReadingGene Therapy for Cardiomyopathy Associated With Friedreich’s Ataxia
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease Post author: Post published:March 25, 2026 Post category: Continue ReadingA Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
A Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary Immunoglobulin A (IgA) Nephropathy at High Risk of Progression Post author: Post published:March 25, 2026 Post category: Continue ReadingA Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary Immunoglobulin A (IgA) Nephropathy at High Risk of Progression
Pembrolizumab and a Vaccine (ATL-DC) for the Treatment of Surgically Accessible Recurrent Glioblastoma Post author: Post published:March 25, 2026 Post category: Continue ReadingPembrolizumab and a Vaccine (ATL-DC) for the Treatment of Surgically Accessible Recurrent Glioblastoma
A Vaccine (Neoantigen-Targeted ppDC) for the Treatment of H3 G34-mutant Diffuse Hemispheric Glioma Post author: Post published:March 12, 2026 Post category: Continue ReadingA Vaccine (Neoantigen-Targeted ppDC) for the Treatment of H3 G34-mutant Diffuse Hemispheric Glioma
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein Post author: Post published:March 1, 2026 Post category: Continue ReadingStudy to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease Post author: Post published:March 1, 2026 Post category: Continue ReadingStudy of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Post author: Post published:March 1, 2026 Post category: Continue ReadingSafety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Post author: Post published:March 1, 2026 Post category: Continue ReadingA Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease Post author: Post published:March 1, 2026 Post category: Continue ReadingTransplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease
