On Wednesday 24 February 2016, Rare Voices Australia -
together with the Mucopolysaccharide & Related Diseases Society Aust. Ltd
(MPS Society) - co-hosted the ‘Standing Tall for Rare Diseases: Spotlight on
Morquio A’ awareness event at Parliament House in Canberra, in the lead up
to Rare Disease Day (29 February 2016).
The event aimed to engage both sides of politics around the
impact of rare diseases, with a particular focus on Morquio A; and the need for
patient access to rare diseases treatments such as enzyme replacement therapy,
VIMIZIM®. The Pharmaceutical Benefits Advisory Committee (PBAC) will consider
VIMIZIM for reimbursement on the Pharmaceutical Benefits Scheme (PBS) listing
when it meets in March (9-11).
L-R: Jason Wood, Sam Cosgrove, Beau Cosgrove, Angus Taylor MP
About Morquio A
Morquio A is a rare but debilitating condition, which
affects just 21 people in Australia. People living with Morquio A – part of the
broader MPS group of diseases – are missing or are unable to produce an
important enzyme, and, as a result, cannot break down certain materials in the
body. These materials eventually build up in tissues, bones, and major organ
systems, leading to potentially serious problems, including heart disease,
skeletal abnormalities, vision and hearing loss, difficulty breathing, and
early death. The average life expectancy is just 25 years of age, with the
primary cause of death in around two thirds of Morquio A patients being
Currently, VIMIZIM is only available through clinical trials
at a select number of hospitals around the country, and those lucky enough to
be on a trial, often have to travel many hours in each direction to access the
treatment, hence the need for it to be made universally available.
About the event
Rare Voices Australia was proud to co-host the parliamentary
BBQ with the MPS Society and Mr John Alexander MP, OAM (Federal Member for
Bennelong, NSW – and Chair of the Parliamentary Friends of Medicines group).
Speakers on the day included:
= Professor Ravi Savarirayan: Clinical
Geneticist at the Murdoch Children’s Research Institute, and contributor to the
development of the ‘International Guidelines for the Management and
Treatment of Morquio A Syndrome’, who spoke about the impact of Morquio A,
and the clinical benefits of VIMIZIM, the only definitive treatment option for
people living with
= Samantha Prior: Acting National Manager
of the MPS Society, who spoke about the role of the Society in supporting
people living with MPS diseases, including Morquio A, as well as the
debilitating impact of the disease – physically, emotionally, psychologically
= Samantha Cosgrove: mother to
five-year-old Beau, from Taralga, NSW, who is currently living with Morquio A
and receiving VIMIZIM through a clinical trial at The Children’s Hospital at
Westmead. Samantha shared the amazing difference she has observed in Beau
during the two years he had received VIMIZIM. On the trial, Sam remarked that “After
the first week on the trial I immediately noticed he had more energy. Last
Christmas he had his swimming lesson, went to the oval and played cricket with
the local kids. He then played at sports club and danced to his favourite
country songs until 8pm. He was up and going the next day with no need to
recover and no complaints of pain.”
Tracey Nelson: mother to three-year-old
Kalten, from Cooma, NSW, who is currently living with Morquio A but does not
have access to VIMIZIM. Tracey spoke about the hope she holds out that families
like hers will one day have access to VIMIZIM, in the same way as the Cosgrove
family, who have seen considerable improvement on treatment.
L-R: Kalten Nelson, Tracey Nelson, Jason Wood, Sam Cosgrove, Beau Cosgrove, Hon Angus Taylor MP, John Alexander MP.
The event attracted the interest of over 40 MPs, Senators
and parliamentary advisers, including: the Hon Stephen Jones MP, Shadow
Assistant Minister for Health and the Hon Angus Taylor MP, Assistant Minister
to the Prime Minister and Federal Member for Hume (Beau Cosgrove’s electorate).
Once the speeches had drawn to a close many attendees stayed
back for more intimate discussions with the speakers. Many in attendance
expressed their support and appreciation of the need to better acknowledge
Morquio A and rare diseases more generally, as well as the need to find the
means to better fund treatment options, such as VIMIZIM.
Of particular note, the Hon. Angus Taylor MP, issued a press
release following the event confirming that he had made a representation to
Health Minister, the Hon Susan Ley MP, on behalf of Beau and his family, to
list VIMIZIM on the PBS.
Beau and Graham Perrett MP.