ataluren

04/01/2016 - "Ataluren is orally bioavailable and was shown to be effective in Cystic Fibrosis (CF). "
01/01/2020 - "Chest computed tomography outcomes in a randomized clinical trial in cystic fibrosis: Lessons learned from the first ataluren phase 3 study."
09/01/2017 - "Ataluren in cystic fibrosis: development, clinical studies and where are we now?"
09/01/2013 - "Response to ataluren was dose dependent, and complete correction was achieved at therapeutic doses currently used in clinical trials for cystic fibrosis. "
04/01/2016 - "Ataluren for the treatment of cystic fibrosis."

01/01/2018 - "Following positive results in ambulatory nmDMD (non-sense mutation Duchenne muscular dystrophy) patients, Ataluren received conditional approval in ambulant nmDMD patients by the EMA in 2014. "
02/01/2014 - "This qualitative study was based on interviews with clinical investigators and parents of sons with Duchenne and Becker muscular dystrophy (DBMD) who participated in the phase IIa or IIb ataluren clinical trial in the United States. "
10/24/2023 - "One of these, ataluren, has received approval in Europe for the treatment of Duchenne muscular dystrophy. "
01/01/2023 - "Ataluren is the only TRID that has been approved for treating patients suffering from a PTC disease, Duchenne muscular dystrophy, but it gives variable readthrough results in cells isolated from patients suffering from other PTC diseases. "
01/01/2022 - "Good response to the late treatment with ataluren in a boy with Duchenne muscular dystrophy: could the previous mild course of the disease have affected the outcome?"

01/01/2018 - "However, larger clinical trials are required to assess the role of Ataluren and its long-term impact on disease progression in non-ambulant nmDMD patients."
08/01/2023 - "Long-term, real-world treatment with ataluren plus SoC delays several disease progression milestones in individuals with nmDMD. "
01/01/2022 - "Results & conclusion: Ataluren plus SoC was associated with a 2.2-year delay in age at LoA (p = 0.0006), and a 3.0-year delay in decline of predicted forced vital capacity to <60% in nonambulatory patients (p = 0.0004), versus SoC. Ataluren plus SoC delays disease progression and benefits ambulatory and nonambulatory patients with nmDMD. "
12/01/2021 - "We can conclude that ataluren appears to stabilize, if not slightly improve, the clinical course of patients with a good safety profile, especially if we consider that the treatment was late for 3/4 patients, at a mean age of 36.6 ± 10.6 years."
10/01/2020 - "Conclusion: These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks. "

12/12/2019 - "Ataluren for the Treatment of Usher Syndrome 2A Caused by Nonsense Mutations."
12/12/2019 - "We provide novel data on the read-through efficacy of Ataluren on a nonsense mutation in the Usher syndrome gene USH2A that causes deaf-blindness in humans. "
05/01/2011 - "We investigated the therapeutic potential of the premature termination codon (PTC) readthrough-inducing drug PTC124 in treating the retinal phenotype of Usher syndrome, caused by a nonsense mutation in the USH1C gene. "

01/01/2021 - "We investigated the safety and efficacy of ataluren in children with nonsense variants causing Dravet Syndrome (DS) and CDKL5 Deficiency Syndrome (CDD). "
01/01/2021 - "Ataluren for drug-resistant epilepsy in nonsense variant-mediated Dravet syndrome and CDKL5 deficiency disorder."
12/01/2023 - "Drug-repurposing approaches for SCN1A-related Dravet syndrome are currently under investigation (i.e., lorcaserin, clemizole, and ataluren). "