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Standing Tall for Rare Diseases: Spotlight on Morquio A

March 2, 2016

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 respiratory failure.

 

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
Morquio A.



=  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 and socially.












= 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.

Category: RVA News
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