Rare Disease Week: In conversation with… Dharmisha, Mum to Sebastian

Join us on Friday 27th February from 12-1pm on Teams for our final event of Rare Disease Week!

Spinal Muscular Atrophy (SMA) is a rare genetic condition that causes progressive muscle weakness to varying degrees. It affects 1 in 100,000 people globally.

We will hear from Dharmisha, Mum to Sebastian, who received gene therapy for SMA Type 1 in 2021. Sebastian was the 6th child in the UK to receive this treatment. Dharmisha will share his diagnosis journey and the impact of gene therapy on the whole family.

This event is open to all healthcare professionals to help develop your understanding of rare diseases.