He should be corrected when clvs treated refractory To plat. ovarion. Cancer??
Please correct me if am wrong!!
But there are other trials in phase III and II... Hope the results come sooner than later..
Why they are doing this vicious selling??
It is unmet needs..it should fly to 50s and higher.
The following splicing modulators have reached clinical stage development:
Nusinersen (formerly, IONIS-SMNRx, ISIS-SMNRx) is a proprietary antisense oligonucleotide developed by Ionis Pharmaceuticals and Biogen which is administered directly to the central nervous system using an intrathecal injection once every few months. It showed promise in phase II trials in infants and children with SMA types 1, 2 and non-ambulant type 3; as of June 2016, it remains in phase III clinical trials.
RG7800 and RG7916 are proprietary small-molecule drugs administered orally developed by PTC Therapeutics and Hoffmann-La Roche. As of June 2016, the clinical development of RG7800 has been put on hold due to animal toxicity while RG7916 is advancing to phase II trials across all patient ages and SMA types.
LMI070 is a proprietary small-molecule drug administered orally, being developed by Novartis. As of June 2016 the drug remains in phase I–II trials in infants with SMA type 1.
Of discontinued clinical-stage molecules, RG3039, also known as Quinazoline495, was a proprietary quinazoline derivative developed by Repligen and licensed to Pfizer in March 2014 which was discontinued shortly after, having only completed phase I trials. PTK-SMA1 was a proprietary small-molecule splicing modulator of the tetracyclines group developed by Paratek Pharmaceutical and about to enter clinical development in 2010 which however never happened.
If it the first effective one ...IONS should go up further and further...to $50 or even $70..