Results 41 to 50 of about 1,505 (151)
A reactivity programmed cascade between carbyne equivalents and allylic benzoates was developed that sequentially orchestrates all three reactivity modes of carbyne equivalents, enabling the rapid installation of three σ‐bonds at a single carbon center and providing efficient, scalable access to structurally diverse spirocyclic ethers. ABSTRACT Carbyne
Jianke Su +5 more
wiley +2 more sources
Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis [PDF]
In a subset of patients with cystic fibrosis (CF), nonsense mutations (premature stop codons) disrupt production of full-length, functional CF transmembrane conductance regulator (CFTR). Ataluren (PTC124) allows ribosomal readthrough of premature stop codons in mRNA.We evaluated drug activity and safety in patients with nonsense mutation CF who took ...
M, Wilschanski +19 more
openaire +2 more sources
(Poly)Borylated Species as Modern Reactive Groups toward Unusual Synthetic Applications
In this review, we spotlight recent breakthroughs in α‐polyboron‐substituted carbon‐centered intermediates (carbanion, carbocation, radical, and carbene) and polyborylated alkenes. By bridging fundamental reactivity with the application potential of these extraordinary species, we hope this review will serve as a roadmap for harnessing these unique ...
Nadim Eghbarieh +5 more
wiley +2 more sources
Congenital aniridia is a rare, pan-ocular disease causing severe sight loss, with only symptomatic intervention offered to patients. Approximately 40% of aniridia patients present with heterozygous nonsense variants in PAX6, resulting in ...
Dulce Lima Cunha +5 more
doaj +1 more source
Die direkte Umwandlung von Alkoholen in tertiäre oder sekundäre Amine in einem Reaktionsschritt unter Verwendung von Nms‐Amiden wird beschrieben. Die Transformation zeichnet sich durch eine breite Toleranz gegenüber verschiedenen funktionellen Gruppen und eine hohe Stereospezifität aus, was ihre Eignung für die Synthese isotopenmarkierter Arzneimittel ...
Jakub Brześkiewicz +3 more
wiley +1 more source
Metabolism and Disposition of Ataluren after Oral Administration to Mice, Rats, Dogs, and Humans [PDF]
Ataluren is a unique small molecule developed for the treatment of diseases caused by nonsense mutations, which result in premature termination of ribosomal translation and lack of full-length protein production. This study investigated the in vivo metabolism and disposition of ataluren in mice, rats, dogs, and humans.
Ronald, Kong +7 more
openaire +2 more sources
In a five-step procedure, 18 new esters ( 20a–i and 21a–i ) of ataluren and its analogs with dihydroartemisinin and zerumbone were synthesized from methyl 3-cyanobenzoate.
Duc Quan Tran +9 more
doaj +1 more source
Clinical and molecular aspects of congenital aniridia – A review of current concepts
Congenital aniridia is a pan ocular disorder characterized by partial or total loss of iris tissue as the defining feature. Classic aniridia, however, has a spectrum of ocular findings, including foveal hypoplasia, optic nerve hypoplasia, nystagmus, late-
Shailja Tibrewal +9 more
doaj +1 more source
The straightforward conversion of alcohols to tertiary or secondary amines employing Nms‐amides in a single operation is described. The transformation features broad functional group tolerance and high stereospecificity, underscoring its utility in the synthesis of isotope‐labeled pharmaceuticals.
Jakub Brześkiewicz +3 more
wiley +1 more source
Background 1,2,4-oxadiazole derivatives exhibited significant anti-cancer activity when they were evaluated, against human cancer cell lines. They also showed anti-inflammatory, analgesic, diabetic, immunosuppressive, α,β3-receptor antagonist ...
Ravi Kumar Bommera +3 more
doaj +1 more source

