The study is a global, randomized, double-blind, placebo-controlled trial testing GLM101 for phosphomannomutase 2 congenital disorder of glycosylation
First and only FDA-approved treatment for seizures associated with CDKL5 deficiency disorder (CDD) in patients two years of age and older
SENS study to assess feasibility, effectiveness and family experience of introducing SMA screening into the NHS newborn screening programme
The transaction includes an upfront payment of $1.55 billion, along with up to $1.1 billion in regulatory and commercial milestone payments
The company is expected to conduct further studies to confirm the long-term efficacy and safety of Vijoice in both adult and paediatric patients
Funding to support clinical development, gene therapy pipeline expansion and GMP manufacturing capabilities
The leading cancer hospital urges carrier screening to prevent thalassemia burden
The acquisition adds VYKAT XR (diazoxide choline), the first FDA-approved therapy for hyperphagia in Prader-Willi syndrome (PWS), a rare genetic disorder, to Neurocrine’s portfolio
Esprit is a multicenter, randomized, placebo-controlled, double-blind study assessing the safety, pharmacodynamics, and preliminary efficacy of S-606001 as a substrate reduction therapy
Achondroplasia, a rare genetic disorder, causes skeletal dysplasia and often increases the risk of muscular, neurological, and cardiorespiratory complications
Subscribe To Our Newsletter & Stay Updated
_256_275.jpeg)
