FDA Grants Orphan Drug Designation to Oligonucleotide Therapeutic for Spinocerebellar Ataxia

Weekly roundup: Designations, data and a dealmaker – Optimum Strategic CommunicationsLink to: Novo Holdings appoints four new members to its Board of Directors further strengthening global perspectives and strategic capabilitiesLink to: Lars Rebien Sørens

Kaerus Bioscience’s lead candidate KER-0193 granted Orphan Drug Designation and Rare Pediatric Drug Designation by U.S. FDA for treatment of Fragile X syndrome (FXS) Kaerus Bioscience announces that its lead candidate KER-0193 has been granted both Orphan Drug and Rare Pediatric Drug Designations for the treatment of Fragile X syndrome (FXS) by the U.S. Food […]

FDA Grants Orphan Drug Designation to Oligonucleotide Therapeutic for Spinocerebellar Ataxia

Cure Rare Disease's CRD-002 receives FDA orphan drug designation, advancing hope for SCA3 treatment with innovative oligonucleotide therapy.