FDA News

The FDA has accepted Sarepta Therapeutics' efficacy supplement for gene therapy SRP-9001, granting it a priority review for the treatment of Duchenne muscular dystrophy, with a decision expected by June 21, 2024.

The chief executive officer of ClearPoint Neuro provided clarity on how the recently approved SmartFrame system can enhance the efficiency of operating room procedures, allowing for flexible workflows and image-based corrections.

Encoded Therapeutics plans to initiate clinical trials assessing its gene therapy candidate ETX101 for the treatment of SCN1A+ Dravet syndrome during the first half of 2024.

VCA-894A, a novel antisense oligonucleotide, exhibits a mechanism of action that specifically targets a cryptic splice site variant within immunoglobulin mu-binding protein 2.

Take a look at some of the most-anticipated FDA pending approvals expected in 2024 that researchers and clinicians in neurology should keep an eye out on.

Takeda's FDA-approved Gammagard Liquid can be used as induction therapy, which includes an induction dose followed by maintenance doses, for adults with chronic inflammatory demyelinating polyneuropathy.

The dihydroergotamine product’s application was supported by phase 1 comparative pharmacokinetic and safety data, and the phase 3 ASCEND trial.

Aruna Bio's investigational treatment, AB126, is now set for a phase 1b/2a clinical trial targeting acute ischemic stroke, anticipated to begin in the first half of 2024.

Takeda's Hyqvia is the only FDA-approved combination of immunoglobulin and hyaluronidase, which makes it a facilitated subcutaneous immunoglobulin infusion, for patients with chronic inflammatory demyelinating polyneuropathy.

The newly FDA-approved AI-powered software platform BrainSee utilizes brain MRI and cognitive assessments to reportedly predict the progression of amnestic mild cognitive impairment to Alzheimer disease in 5 years.

Catch up on any of the neurology news headlines you may have missed over the course of January 2024, compiled all into one place by the NeurologyLive® team.

Months after the FDA cleared a phase 2 study in myasthenia gravis, KYV-101 is set to be assessed in a diverse cohort of refractory progressive multiple sclerosis.