Neurological Symptoms Persist After SARS-CoV-2 Infection
Memory impairment and decreased concentration most prevalent symptoms at six-month follow-up
Miscellaneous Neurodegenerative Disorders
Amvuttra Approved for Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis
Amvuttra is a subcutaneously-administered RNAi therapeutic designed to block the production of wild-type and mutant transthyretin protein.
Fenfluramine Seems Effective for Lennox-Gastaut Syndrome
Reduction in frequency of drop seizures greater with fenfluramine versus placebo
Medicare Will Only Cover Aduhelm for Patients in Clinical Trials
Decision is meant to protect patients while gathering more clinical data on whether the medication actually helps slow declines in thinking
Enrollment Paused in Trials Evaluating BCX9930 for Complement-Mediated Diseases
BCX9930 is an investigational oral Factor D inhibitor.
Memory Deficits Seen in Individuals With ‘Long COVID’
Deficits increased with severity of ongoing self-reported symptoms; severity of initial illness did not influence later cognitive performance
Elamipretide Gets Orphan Drug Status for Friedreich Ataxia
Elamipretide has been shown to improve frataxin levels in Friedreich ataxia patient-derived cells, and improve motor and cardiac function in an animal model of Friedreich ataxia.
Gene Therapy for Canavan Disease Receives Multiple FDA Designations
The gene therapy candidate is designed to deliver the ASPA gene directly to the oligodendrocyte cells in the fluid surrounding the brain and spinal cord.
Gene Therapy Candidate Gets Orphan Drug Status for Adrenomyeloneuropathy
The Company will initiate a randomized, controlled phase 1/2 trial to evaluate the efficacy and safety of SBT101 in patients with AMN in the second half of 2022.
Zilucoplan Improves Myasthenia Gravis Symptoms in Phase 3 Trial
Zilucoplan is a synthetic, macrocyclic peptide inhibitor of complement component 5.
