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Victoria Johnson
Articles
PTC’s AADC Deficiency Gene Therapy Leads to Durable Developmental Improvements
October 04, 2021
Article
Children with AADC receiving treatment developed motor function and cognitive skills that persisted through up to 10 years of follow-up.
CAP-1002 Meets Primary End Point in DMD in HOPE-2 Trial
October 01, 2021
Article
Patients with Duchenne muscular dystrophy receiving the treatment had a decline in Mid-level Performance of Upper Limb scores in the phase 2 trial of the cell therapy.
SMA Gene Therapy Cleared to Proceed Following FDA Clinical Hold
August 09, 2021
Article
In addition to the uplifted phase 1 study, Novartis also initiated the phase 3 STEER study, which will evaluate OAV-101 in treatment-naive patients with SMA type 2 aged between 2 and 18 years old.
Sleep Profiles May Predict Cognitive Decline in Alzheimer Disease
June 17, 2021
Article
While the relationship between sleep and cognition needs to be further studied, sleep-based interventions may be a future approach to help prevent cognitive decline.
Vamorolone Efficacious in Treatment of DMD, Superior to Prednisone in Safety
June 16, 2021
Article
Santhera and ReveraGen announced findings of the phase 2b VISION DMD study of the investigational therapy.
Gene Therapy Studies Underway in Parkinson Disease
June 15, 2021
Article
Bayer provided an update on their phase 1 and 1b studies of dopaminergic neurons and GDNF gene therapy, respectively, for the treatment of PD.
Solriamfetol Efficacious, Easily Titrated in Patients Initiating Treatment for Narcolepsy
June 12, 2021
Article
Results from the START study of medication switching in narcolepsy were presented at the 2021 SLEEP Annual Meeting.
FT218 Efficacious in Treating Narcolepsy Subtypes
June 10, 2021
Article
Data presented at SLEEP 2021 also showed the treatment was associated with significant weight loss and reduction in BMI compared to placebo.
Rimegepant Achieves Migraine Prevention After 1 Week of Treatment
June 09, 2021
Article
A post-hoc analysis of the phase 2/3 trial of rimegepant revealed early and sustained efficacy compared to placebo, with significant reductions in weekly migraine days.
New Migraine Burden Index Proves Accurate, Strongly Correlates With Patient Satisfaction
June 09, 2021
Article
Investigators conducted a 9-month study that demonstrated the correlation of the MBI, calculated as the sum of headache days times the maximum intensity of headache on each headache day, with satisfaction of migraine status.