FDA Gives ET-101, Gene Therapy for ALS, Orphan Drug Status
ET-101, Eikonoklastes Therapeutics’ experimental gene therapy for amyotrophic lateral sclerosis (ALS), has been designated an orphan drug by…
Articles by Vanda Pinto, PhD
EU Committee Urges Orphan Drug Status for Clene’s CNM-Au8 for ALS
Orphan drug status has been recommended in the EU for Clene Nanomedicine’s CNM-Au8, an oral…
Chest Pain Medication May Help Ease Muscle Cramps in ALS
An approved medication for heart-related chest pain reduced the frequency and severity of muscle cramps in people with amyotrophic…
Vocal Cord Spasms in ALS May Be Triggered by Excessive Saliva
Although not thought common, laryngospasm — the sudden contraction of the vocal cords that makes speaking and breathing temporarily difficult…
Phase 2 Trial of AIT-101 in Patients With C9orf72 Mutations Enrolling
AI Therapeutics has launched a Phase 2 clinical trial into AIT-101 (apilimod dimesylate) as a potential oral treatment for…
Phase 2 Safety Trial of AT-1501 Nears Full Enrollment of Patients
A Phase 2 study of the investigational antibody AT-1501 in treating amyotrophic lateral sclerosis (ALS) has completed patient enrollment in three…
Patient Trials Planned or Underway for 2 Oral ALS Therapies, Denali Says
Early trials in healthy adults supported the safety and tolerability of two potential oral therapies for amyotrophic lateral sclerosis…
Clene Soon Will Complete Phase 2 Trial Testing CNM-Au8
Note: This story was updated Sept. 21, 2021, to note that 18% of patients, not 80%, experienced increases in…
Dosing Begins in Phase 2 Trial of AL001 in ALS With C9orf72 Mutations
A first patient has been dosed in a Phase 2 clinical trial testing Alector’s investigational antibody AL001 as a…
Clene Boosts Production Capacity of CNM-Au8
Clene, and its wholly owned subsidiary Clene Nanomedicine, have signed two lease agreements that will more than quadruple the…