The study describes the genetic basis of ACHM
segregating in an autosomal recessive pattern in two families (PKCN02 and PKCN-07) of Pakistani origin (Figure 1A, 1B).
has drafted this set of criteria to help guide hospital credentialing committees when it comes to hyperbaric training of physicians,” states Dr.
, Inventario Frentes de Guerra, Tendencia GPP, Caja IV, Folder 1, Expediente 14.943.
MeiraGTx announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the FDA have granted rare pediatric disease designation to the company's gene therapy product candidate AAV-CNGA3 for the treatment of patients with achromatopsia, or ACHM
, due to mutations in the CNGA3 gene.
FDA has granted Fast Track designation for its AAV-CNGB3 gene therapy product candidate for the treatment of achromatopsia, or ACHM
, caused by mutations in the CNGB3 gene.
is an inherited retinal disease that severely limits a person's sight by preventing cone photoreceptors in the eye from functioning.