https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/brc-news/spinraza-first-treatment-spinal-muscular-atrophy-receive-fda-approval/
Spinraza: the first treatment for spinal muscular atrophy to receive FDA approval
30 Mar 2017, 11:28 a.m.

At the end of 2016, the American Food and Drug Administration (FDA) granted approval for the use of Spinraza (nusinsersen) for the treatment of spinal muscular atrophy (SMA) in paediatric and adult patients. This is unprecedented for the SMA community witnessing the first approved drug for this disease.
The FDA approval was based on positive results from multiple clinical studies in more than 170 patients. GOSH was the highest UK recruiter for the first study, ENDEAR, a Phase 3, controlled study evaluating Spinraza in infantile-onset, which took place in the BRC-supported Somers Clinical Research Facility.
The interim analysis of the data from ENDEAR, (previously reported in the December BRC newsletter) was considered throughout the approval process. In particular, patients with infantile-onset SMA recruited to the ENDEAR study and treated with Spinraza, achieved and sustained clinically meaningful improvement in motor function compared to untreated study participants. Furthermore, a greater percentage of patients on Spinraza survived compared to untreated patients. In open-label studies, some patients also achieved unexpected milestones such as ability to sit unassisted, stand or walk.

New study pinpoints why some transplanted kidneys are rejected
Researchers at University College London (UCL), the Wellcome Sanger Institute and the University of Cambridge combined technology that determines the genetic or RNA sequence of individual cells with powerful 3D imaging to look at lymphatic vessels

Teenager off to university after life-changing gene therapy
A teenager with a rare, life-limiting genetic condition is off to university after receiving a gene therapy that changed his life.

Toddler doing well after receiving newest gene therapy available on NHS
A toddler with a life-limiting and life-threatening rare disease is the youngest to be treated with the newest gene therapy available on the NHS at GOSH.

Join us in the countdown to Rare Disease Day 2025
Rare Disease Day takes place annually on the last day of February and is a global opportunity to increase awareness and advocate for those living with rare conditions.