{"id":3505,"date":"2025-06-13T20:45:29","date_gmt":"2025-06-13T20:45:29","guid":{"rendered":"https:\/\/life4hsp.com\/?p=3505"},"modified":"2025-06-23T11:05:38","modified_gmt":"2025-06-23T09:05:38","slug":"first-trial-of-new-gene-therapy-for-hsp-spg47-approved","status":"publish","type":"post","link":"https:\/\/life4hsp.com\/en\/first-trial-of-new-gene-therapy-for-hsp-spg47-approved\/","title":{"rendered":"First trial of new gene therapy for HSP (SPG47) approved"},"content":{"rendered":"

There is hopeful news for people with HSP: the US health authority FDA has approved an initial trial of a new gene therapy for a rare form of hereditary spastic paraplegia, namely SPG47.<\/span><\/p>\n

What is SPG47?<\/b><\/h3>\n

SPG47 is a rare, hereditary form of HSP where children show symptoms at an early age, such as muscle stiffness, difficulty walking, developmental delays, and speech problems. The cause lies in a mutation in the SPG47 gene, which prevents an important protein in brain cells from functioning properly. Currently, there is no treatment available.<\/p>\n

What will happen now?<\/b><\/h3>\n

A biotech company, BlackfinBio<\/a>has been granted permission to begin the first study involving children with SPG47. The trial will take place at a specialized hospital in Boston (USA). The goal is to determine whether the treatment is safe.<\/p>\n

The treatment involves a one-time injection into the cerebrospinal fluid containing a healthy piece of DNA. This healthy gene should enable the child\u2019s cells to produce the correct protein again.<\/p>\n

Why is this important?<\/b><\/h3>\n