AstraZeneca Marks Milestone in SA Healthcare With Launch of Rare Disease Treatments for Children

The event included a panel discussion with experts from fields such as neuro-oncology, neurology, plastic surgery, and genetics.

Panellists highlighted the vital need for coordinated, specialist-driven care and robust collaboration across public and private sectors to ensure that patients receive integrated support at every stage of their journey.

Dr Nkuli Boikhutso, CEO of Nelson Mandela Children’s Hospital, stated: “We are honoured to be the site of this important milestone.

"AstraZeneca’s commitment to introducing new treatments for rare diseases offers hope for our young patients and aligns with our vision to provide world-class, specialised care for all children, regardless of their background.”

Rare diseases

Around the world, more than 400 million people are affected by over 10,000 known rare diseases, yet the vast majority of these conditions have no approved treatments.

For many rare disease patients and their families, this often results in years of uncertainty, misdiagnosis, and limited or difficult treatment options.

AstraZeneca is dedicated to transforming this landscape by introducing advanced therapies, supporting earlier and more accurate diagnoses, and fostering multidisciplinary collaboration.

“AstraZeneca’s entry into the rare disease space in South Africa is both a proud and hopeful moment for us,” said Deepak Arora, African cluster country president at AstraZeneca.

“Building on decades of pioneering rare disease science, we are committed to expanding access to transformational medicines for conditions such as NF1, PNH.

“This marks a significant milestone in our mission to improve the lives of patients living with rare diseases, especially children who have had very few options until now.”

AstraZeneca’s rare disease strategy in Africa is anchored in three key priorities: accelerating access to vital medicines, driving innovation through research and development, and expanding its global reach to make meaningful change.