Sanfilippo Syndrome Type A (MPS IIIA)
Also known as: Sanfilippo Syndrome A / Sanfilippo Syndrome Type A / Sanfilippo A / Mucopolysaccharidosis IIIA / Sanfilippo Disease Type A / MPS IIIA / Mucopolysaccharidosis Type III A / Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome / Mucopolysaccharidosis Type 3 A / Mucopolysaccharidosis Type IIIA / Sanfilippo mucopolysaccharidoses / Mucopolysaccharidosis, MPS-III-A (disorder) / MPS III A
| Drug | Drug Name | Drug Description |
|---|
| Drug | Drug Name | Target | Type |
|---|
| Drug | Drug Name | Phase | Status | Count |
|---|---|---|---|---|
| DB16367 | Autologous peripheral blood-derived CD34+ cells | 1 / 2 | Active Not Recruiting | 1 |
| DB16784 | Rebisufligene etisparvovec | 1 / 2 | Terminated | 1 |
| DB15451 | SOBI-003 | 1 / 2 | Completed | 2 |
| DB16264 | Olenasufligene relduparvovec | 2 / 3 | Active Not Recruiting | 1 |
| DB16784 | Rebisufligene etisparvovec | 2 / 3 | Active Not Recruiting | 1 |