4 results
We propose a phase II study (intervention) in patients with severe generalized recessive dystrophic EB receiving reduced toxicity conditioning chemotherapy followe by cord blood transplantation with co-infusion of mesenchymal stromal cell units.…
The primary endpoint of this feasibility study is the technical success of positioning the SLIDE device to verify the speed and ease of use without unexpected device related adverse events. A technical success is defined that the SLIDE device was…
To investigate efficacy of ASP6294 in female subjects with Bladder Pain Syndrome/Interstitial Cystitis (BPS/IC).To investigate safety and tolerability of ASP6294 in female subjects with BPS/IC.To investigate the pharmacokinetics and pharmacodynamics…
The purpose of this study is to characterize the safety and tolerability of OAV101 IT in participants who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®). The data from this study will expand on the data generated…