VJToday /Features

FDA grants accelerated approval for SRP-9001 as the first ever gene therapy for Duchenne muscular dystrophy

On 22nd June, 2023, the U.S Food and Drug Administration (FDA) granted accelerated approval to Sarepta Therapeutics’ SRP-9001 (ELEVIDYS, delandistrogene…

VJToday
1 month

ENRICH trial demonstrates efficacy of minimally invasive surgery in intracerebral hemorrhage  

ENRICH is a two-arm, multicenter, randomized, adaptive clinical trial assessing the effectiveness of minimally invasive trans-sulcal parafascicular surgery (MIPS) for…

VJToday
2 months

Trofinetide approved by FDA as first ever treatment for Rett syndrome

On March 10th 2023, the Food and Drug Administration (FDA) approved trofinetide in the treatment of Rett syndrome (RTT), making…

VJToday
4 months

First ever targeted treatment for SOD1-ALS: FDA approve tofersen

On April 25th, 2023, the U.S. Food and Drug Administration (FDA) approved tofersen (BIIB067) for the treatment of amyotrophic lateral…

VJToday
4 months

Newborn screening improves health outcomes in infants with spinal muscular atrophy

Spinal muscular atrophy (SMA) is a severe genetic disease associated with mutations in the SMN1 gene (SMA5q). The pathological characteristics…

VJToday
7 months

Relaunching the GENERATION HD trial: how will tominersen fare with younger, milder Huntington’s disease patients?

Following the termination of the GENERATION HD1 trial in March 2021, Roche have now announced plans for a new Phase…

VJToday
8 months

FDA approves ublituximab for relapsing multiple sclerosis

The Food and Drug Administration (FDA) approved ublituximab for the treatment of relapsing forms of multiple sclerosis (MS), making it…

VJToday
8 months

Active seizures worsen clinical and mortality outcomes for patients with dementia

New data presented at the American Epilepsy Society (AES) 2022 annual meeting has demonstrated that the presence of active seizures…

VJToday
8 months

MR CLEAN-LATE results presented at the World Stroke Congress 2022

The MR CLEAN-LATE prospective, randomized study of over 500 patients with acute ischemic stroke showed that endovascular thrombectomy (EVT) provides…

VJToday
10 months

AMX0035 receives FDA approval for ALS

On September 29, 2022, the Food and Drug Administration (FDA) approved AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS),…

VJToday
11 months
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