UN Event For Rare Disorder SMA: Leading Advocates Urge Government To Act For Those Living With The Condition In NZ

October 29, 2021

All change starts with awareness and today we had the privilege to be part of a powerful United Nations event to raise awareness of a rare disorder called Spinal Muscular Atrophy (SMA). The opening address was provided by H.E. Mr. Sergiy Kyslytsya, Permanent Representative of Ukraine to the United Nations and Mac Reynolds from Imagine Dragons, who also shared their uplifting music with the delegates.

The event was attended by senior UN representatives, including the President of the United Nations General Assembly, the President of ECOSOC and representatives of diplomatic missions in New York, major UN entities such as UNICEF, WHO, UNDP and UNFPA, pharmaceutical company representatives, and patient advocacy groups.

Rt Hon. Helen Clark, who is Board Chair of the Partnership for Maternal, Newborn and Child Health (PMNCH), was one of the special guests, and emphasised the need to not leave those with SMA behind in the global journey towards sustainable development goals. Helen noted that rare diseases affect 300 million people worldwide and a community of this scale demands and deserves global attention.

During the event, the importance of early detection via newborn genetic screening and access to responsive treatment were shared. The reality that approximately 1 in 40 people will unwittingly be carriers of the SMA gene illuminates the enormous benefits of prevention via carrier screening or pre-implantation testing for IVF. Some countries such as Australia recently announced measures to offer such support and Helen Clark applauded the Ukraine for their rapid decision  to provide newborn genetic screening and treatment for SMA by end of this year.

“Global solidarity and shared learnings between Member States provides opportunities for bold action that could address the global discrepancies of access to treatment and care” says Lisa Foster, Chief Executive of Rare Disorders NZ. “A vision without action is just a pipe dream yet luckily there is already a roadmap for New Zealand to follow with most developed nations having National Frameworks and action plans in place. There is also current advocacy for the adoption of a UN resolution to address the challenges of persons with rare diseases. If adopted, it would be an amazing first step”, Lisa states.

Effective advocacy on a global scale can accelerate access to treatment and a chance for a normal life or better quality of life for patients suffering from SMA and children about to be born with SMA. (main objective of event)

“The challenge in front us now, as we talk about achieving Universal Health Care, is to make such services more widely and equitably available around our world”, stated. Helen Clark.

Fiona Tolich, advocate for Spinal Muscular Atrophy and a trustee with Patient Voice Aotearoa, shared her powerful journey as a patient living with SMA in New Zealand. Fiona highlighted the importance of introducing early detection via a newborn screening programme, which would provide children with a 70% chance of showing no clinical signs of the disease at all if treated.

Sadly, although three treatments for SMA have been developed, and one of these, SPINRAZA has some form of reimbursement in 64 countries and a high priority status, there is no funded access to any treatment for SMA in New Zealand. Fiona spoke about the disparity between Australia and New Zealand, which has rendered many New Zealand citizens medical refugees. Her closing remarks explained that New Zealand needs a system that does not discriminate based on the condition you are born with and that uses a fair and equitable full societal evaluation when making funding decisions.

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