Haris was born in August 2018, a healthy happy baby. Unfortunately Haris did not meet the midwifes’ expected milestones and was referred to specialists for further tests. Whilst awaiting the results for tests Haris was rushed to hospital in February 2019 and diagnosed with Spinal Muscular Atrophy Type 1. The biggest killer of toddlers under 2 in the U.K. With no treatment available in England at the time Haris was given just months to live.
Nusinersen better know as Spinraza was available in the majority of EU countries at the time including on the NHS in Scotland. It was not available to access on the NHS England. A successful campaign was launched by the family with support from many well known faces. Local MP Mike Kane and London MP Lynn Brown made strong representations on behalf of the family. Treatment was approved on May 15th 2019 and Haris received his first dose on June 8th at Royal Manchester Children’s Hospital.
Zolgensma the worlds most expensive drug at £1.8 million was approved by the NHS on March 8th 2021 after successful trials in U.S. & across Europe. The full qualifying criteria is due to be published by NICE (National Institute for Health & Care).
The benefits of Zolgensma for Haris are described as remarkable and there is strong evidence to support significant milestones being achieved by SMA Type 1 patients.