NVS reports new safety and efficacy data from the phase III program for its investigational OAV101 IT gene therapy for SMA patients.

Axsome's phase III FOCUS study, evaluating solriamfetol for treating attention deficit hyperactivity disorder, meets its primary and key secondary...

Axsome's phase III FOCUS study, evaluating solriamfetol for treating attention deficit hyperactivity disorder, meets its primary and key secondary...

UNITY Biotechnology stock loses 29% as the mid-stage DME study of the lead candidate, UBX1325, fails to achieve statistical significance for the...

One of two of Opthea's phase III trials for a common eye disease has failed to meet its primary endpoints, ...

One of two of Opthea's phase III trials for a common eye disease has failed to meet its primary endpoints, ...

Despite the reported death, Sarepta maintains that its gene therapy has a favorable 'benefit-risk profile.'

Despite the reported death, Sarepta maintains that its gene therapy has a favorable 'benefit-risk profile.'

Esperion plans to begin two phase III studies on bempedoic acid alone and in combination with ezetimibe in pediatric patients with familial...

Esperion plans to begin two phase III studies on bempedoic acid alone and in combination with ezetimibe in pediatric patients with familial...