Rose is an associate editorial director at The American Journal of Managed Care® (AJMC®).
She has a BA in journalism & media studies and Spanish from Rutgers University. You can connect with Rose on LinkedIn.
Quantitative Magnetic Resonance Outcomes Suggest Gene Therapy Slows DMD Progression
Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients with Duchenne muscular dystrophy (DMD) based on muscle quantitative magnetic resonance measures.
Insights Into Iptacopan in C3 Glomerulopathy: Carla Nester, MD, MSA, FASN
Carla Nester, MD, MSA, FASN, is coinvestigator for the ongoing APPEAR-C3G trial (NCT04817618), data from which were used to support the FDA’s recent approval of iptacopan (Fabhalta; Novartis) in complement 3 glomerulopathy (C3G).
The Value of Newborn Screening for DMD: Barry Byrne, MD, PhD
Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for patients with Duchenne muscular dystrophy (DMD).
Future Research Directions for Revakinagene Taroretcel in MacTel
Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, spoke to the current state of research on revakinagene taroretcel in macular telangiectasia type 2 (MacTel), as well as the importance of patient groups for the rare condition.
How Revakinagene Taroretcel Can Benefit Patients With MacTel: Dr Charles Wykoff
The approval of revakinagene taroretcel (Encelto; Neurotech) addresses a significant unmet need for patients with macular telangiectasia type 2 (MacTel), clinical investigator Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, said.