- The US FDA has granted ODD for branaplam in HD. In preclinical trials, branaplam demonstrated a reduction in levels of the mutant huntingtin protein. Additionally, the therapy showed a reduction in huntingtin mRNA in SMA patients
- Novartis expects to initiate the P-IIb study for branaplam in HD patients in 2021
- Branaplam (qw, PO) is an RNA splicing modulator, currently under investigation for the treatment of SMA
Click here to read full press release/ article | Ref: Novartis | Image: Pfarma
The post Novartis’s Branaplam (LMI070) Receives the US FDA’s Orphan Drug Designation for Huntington’s Disease first appeared on PharmaShots.