Inc. (Nasdaq: ACST) (Acasti or the Company), a late-stage, biopharma company advancing GTX-104, its novel injectable formulation of nimodipine that addresses high unmet medical needs for a rare ...

GTX-104 is a clinical stage, novel, injectable formulation of nimodipine being developed for intravenous (IV) infusion in aSAH patients to address significant unmet medical needs. The unique ...

Acasti Pharma Inc. (Nasdaq: ACST) (Acasti or the Company), a late-stage, biopharma company advancing GTX-104, its novel injectable formulation of nimodipine ...

Based on a clinical trial design and endpoints that ... in the treatment of the neurological disease spinocerebellar ataxia (SCA). Troriluzole is a prodrug, a compound that converts into an ...

A clinical trial is one that involves human participants and seeks to answer specific questions about a type of medical intervention. This can be a drug or other type of treatment, such as nutritional ...

Hereditary hemorrhagic telangiectasia (HHT) is characterized by extensive telangiectasias and arteriovenous malformations. The primary clinical manifestation is epistaxis that results in iron ...

This past May, Larimar Therapeutics, a clinical-stage biotechnology company focused on developing treatments for rare diseases, announced that its lead compound for Friedreich's Ataxia, Nomlabofusp, ...

Although most patients with PAVMs may be entirely asymptomatic, PAVMs can induce a series of complications involving the neurological, cardiovascular, and respiratory systems that can lead to ...

Purpose Patients with non-proliferative macular telangiectasia type 2 (MacTel) have ganglion cell layer (GCL ... A total of 1585 OCT volume scans were automatically segmented and manually reviewed for ...

Bayesian analysis combines previous information (represented by a mathematical probability distribution, the prior) with information from the study (the likelihood function) to generate an updated ...

Clinical trials of this drug have shown ... Susan Milman, from Euro-Ataxia, 8 emphasised how patients and families “need information about how to live with the condition and how not to let the ...