A study to evaluate the long-term efficacy and safety of AT-GTX-501 in patients with CLN6 Batten disease, who have received AT-GTX-501 in a previous study. A study to evaluate the long-term efficacy and safety of AT-GTX-501 in patients with CLN6 Batten dis ...
LONG-TERM FOLLOW-UP OF AT-GTX-501 SCAAV9 GENE TRANSFER IN SUBJECTS WITH CLN6 BATTEN DISEASE
Patients with variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease), a subset of patients with CLN6 Batten disease. MedDRA version: 23.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Patients with variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in t ...
Product Name: scAAV9.CB.CLN6 Product Code: AT-GTX-501 INN or Proposed INN: Not yet assigned Other descriptive name: Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene Product Name: scAAV9.CB.CLN6 Product Code: AT-GTX-501 INN or Proposed INN: Not yet assigned Other descr ...