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.