Please click on this link to see the Government's
Guidelines on the use
of Elaprase through the Life Saving Drugs Program.
Please click on this link to read the
Autumn 2009 Edition of Linking Hand
An excellent MPS
Information Session was held at the Royal Children's Hospital
in Melbourne on 15 March 2009. The guest speaker was Professor Lorne
Clarke from Canada, an expert in Lysosomal/MPS Diseases. We will
keep you informed of any other similar opportunities that arise.
The Australian Government
announced in early February 2009 arrangements for the new advisory
structure to support Australia’s
National
Medicines Policy.
Please click on the link below for more information:
http://www.health.gov.au/internet/ministers/publishing.nsf/Content/mr-yr09-nr-nr012.htm?OpenDocument&yr=2009&mth=2
This is a funding scheme which can offer
financial assistance to individuals and families affected by
Mucopolysaccharidoses, and to allow a fair and equitable way to support
families attending the biannual National conferences.
Grants can be applied for anywhere up to a maximum of $500.
Examples of potentially eligible costs are special equipment, medical aids,
education, counselling and respite. A special funding round is held prior
to a conference.
A review committee has been setup to review applications.
Application forms will shortly be added to this website for downloading or a
form can be posted to you, in which case, please contact the office and request
an application form on 02 94768411 or email
secretary@mpssociety.org.au.
These packs will explain the criteria for applicants and more details about the
program.
1st Asia-Pacific Lysosomal Conference and 12th National Australian
MPS Conference
Christchurch 20th – 23rd November 2008
After 2 years of
planning, fundraising and gathering together some of the world’s leading experts
in Lysosomal diseases, the excitement began to build with families and
professionals looking forward to coming down under to meet, share information
and provide a knowledge base that is difficult to gain in our part of the world.
What started out as an Asia-Pacific conference became an International meeting
with Professionals and other support group leaders coming from, Germany, Norway,
USA, Japan, Hong Kong, United Kingdom, Israel, Canada, Australia and New
Zealand.
The opening of conference was held on Thursday 20th Nov, at 6pm on the Village
green at the stunning Chateau on the Park, as the sun began to set.
Delegates were welcomed by KoTane the Cultural experience group from
Christchurch. Prof John Hopwood from Adelaide was chosen as the conference chief
and Bowen Oliver MPS6 represented all those affected by Lysosomal Disorders.
Peter Joyce from the Christchurch school of medicine opened the conference with
John Forman LDNZ and David Oliver from MPS Aust. Ltd, responding. It was
wonderful to finally see everyone gathered together talking, meeting for the
first time and renewing old friendships.
Friday began the first full day of the conference program covering a very wide
range of topics. The Plenary addresses were presented by Prof. John Hopwood
Australia, Steve Walkley USA and Brian Winchester, UK. Their presentations gave
an overview of Lysosomal Diseases, The challenges of LSD’s in particular the
problems faced in trying to cross the blood brain barrier the and central
nervous system, and Biochemical and Molecular diagnosis of Lysosomal Diseases.
Jonathan Cooper presented a very interesting presentation on Batten disease in
mouse models and the ability to study the pathogenesis at multiple stages of
disease progression which is not feasible in the human disorder. Kim Hemsley
presented a wonderful presentation on “What’s happening in Sanfilippo Research”?
Families whose children are affected with this disease have waited a longtime
for research to progress to a level where there are a number of things happening
that could in time benefit their disorder.
Ed Wraith from the UK discussed Existing Therapies their benefits and
limitations. Bone Marrow transplants have been available for some of the LSD’s
for a number of years but is only truly effective if the child is diagnosed
early and transplant is done early. Enzyme replacement therapy is a safer
approach but is ineffective in disorders affecting the brain as the enzyme is
unable to cross the blood brain barrier. ERT is very expensive and is not
available to patients from countries that have other competing health care
needs.
Later in the day we looked at emerging therapies for Morquio A, Alpha
Mannosidosis, Pompe disease. It is exciting to see other Lysosomal Diseases
getting to the point of clinical trials and clinic use of these life saving
drugs, the day closed with a Poster viewing session.
The next day we looked at the clinical management of these diseases and looked
at other therapies out on the fringe. After lunch the conference was broken down
into workshops for families and professionals. Each workshop had three short
presentations and then open discussion, where families could ask questions and
share information on how they manage their particular disorder.
The conference closed at 5pm allowing everyone to have a break and prepare for
the Gala Dinner celebrating the 25th Anniversary of the Australian MPS Society.
The gala dinner was an opportunity for everyone to get dressed up and enjoy some
good old Kiwi hospitality. KoTane came back and entertained us with a haka, poi
dance, stick games and interacted with the audience. It was wonderful to see
everyone taking part and having some fun. Later in the evening LDNZ honored Prof
David Sillence Westmead Children’s hospital Sydney, Australia and Prof. Bob
Jolly, Massey University, Palmerston North, New Zealand, with Life time awards.
Prof Sillence (Australia) has played a very special role in the lives of many
New Zealanders affected by LSD’s. In the 1990’s he would come to New Zealand and
hold specialist genetic clinics at a time when this service was not available to
New Zealand families. He would also bring NZ families to Sydney for full medical
work ups. His compassion and care of all families affected by these diseases has
been truly outstanding. Dr Jim McGill (Australia) also honored David and
presented a small presentation of his life.
Prof Bob Jolly (New Zealand) has spent his life working on Lysosomal Diseases
and in the early days discovered Alpha Mannosidosis in Angus cattle in New
Zealand. These discoveries lead to an understanding of the human disease and
eventually lead to successful Bone Marrow transplants being done on patents who
were diagnosed early. His work has also been in the field of batten disease and
more lately in Sanfilippo disease.
LDNZ presented the Australian MPS Society with their 25th Anniversary cake and
David Oliver did a fantastic presentation outlining the history of the MPS
Society. There was a photo call for the Past presidents of the MPS Society and
we were thrilled to have Denise Law, Teresa Llewellyn and Wendy Boon present on
the night.
Sunday the last day of conference bought us all together again to discuss the
issues of New Born screening and Access to Medicines. New Born Screening looked
at applying the science, applying it to the program and the ethical issues of
new born screening.
And finally Access to medicines the Australian experience and what are we going
to do in New Zealand to get access to Enzyme Replacement therapies. LDNZ has
much work to do in this area and the time is now right for us to start the
advocacy for our New Zealand patients.
The conference was very nicely summed up by Dr Dianne Webster with David Oliver
closing the conference and inviting everyone to join together for the last time
with a combined family/professional lunch.
Jenny Noble
Conference Convenor
Secretary/Treasurer
Lysosomal Diseases New Zealand
Please also see a report and photos in the latest
Linking Hand
Please click on this link to read reports on the
conference in an excerpt from the
UK Winter
2008 MPS Magazine
Click on this link to see some
conference photos
    |
|
Lysosomal Diseases New Zealand was very proud to
host the 12th National Australian MPS and Related Diseases conference
and the 1st Australasian Lysosomal conference in Christchurch New
Zealand, November 20th-23rd 2008. Click on this link to
view
LDNZ
Conference information. |
Excerpt from email from Laura Moldovan to Greg Moran (Treasurer of the
Australian MPS Society):
While conducting some research in worldwide MPS Societies
I decided to write to you about what we think would be a valuable
addition the society’s library of knowledge in mucopolysaccharidosis
type II (MPS II, Hunter disease).
For the past 2 years our group was involved in creating
an educational software project: The Hunter disease eClinic, designed to
meet the needs of clinicians, biologists, geneticists, biochemists, and
other healthcare professionals who are interested in independent
learning about a rare, genetic metabolic disease, mucopolysaccharidosis
type II (MPS II, Hunter disease).
It is a pilot project, first of its kind as far as we
know, and so far met with a lot of enthusiasm in the scientific
community being characterised as a valuable resource that covers all the
aspects and science regarding this disease.
The software was launched in early summer, for the 10th
International Symposium on Mucopolysaccharides and Related Diseases in a
both CD (Windows/Mac) and web format. The web version can be accessed at
our group's web-site under "NEW: Educational Resources" on our group
home page:
http://www.sickkids.ca/lysosomalresearchgroup/
The direct link to the educational software is:
http://www.sickkids.ca/lysosomalresearchgroup/contents/Hunter/Hunter.asp
however the user will have to be warned that they need “Flash Player” to
be installed on local machine in order to be able to view the program
and also to not use the browser back button.
Warm regards,
Laura Moldovan, Clinical Research Analyst,
Computer Teaching SW
http://www.sickkids.ca/lysosomalresearchgroup/
The Hospital for Sick Children
Clinical and Metabolic Genetics
525 University Ave
10th floor, Suite 1045
Toronto, Ont M5G 2L3, Canada
Please note that there is an international MPS Forum site
found at
http://www.mpsforum.com/forum/ which is the MPS/ML Forum Dot Com -
An Internet Gathering Place for MPS/ML Families.
This site has no connection with the Australian MPS
Society. We cannot guarantee or vouch for any information or view
expressed in the forum and have no control whatsoever over its content
or use. Any use of the forum is at the users own risk.
BioMarin Announces Program for ERT for
Treatment of MPS IVA - Morquio A Syndrome
Please click on this link to read
BioMarin's
Announcement.
Please keep posted, we will provide these results as soon
as authorised to do so.
http://www.speakwell.com/well/2004winter/simon.php Please click on
this link to read Simon Ibell's story
The AGM for 2008 was held on 13 April 2008 in Sydney.
The Board and Committee are delighted that David Oliver has agreed to
take on the role of President for the next year.
The Committee offers its very grateful thanks to
Vanessa Ede-Scott, Lillian Lodewyk and Louise Jessop who have stepped
down from the Committee. The Committee welcomes Nicole Millis to
the Committee.
To increase awareness of Mucopolysaccharide and Related
Diseases, the international MPS Community has decided that we should
annually hold an Awareness Day on 15th May. The first ever
Awareness Day was celebrated on 15 May 2007. Those on
our mailing list were sent bumper stickers and postcards to provide
to family, friends and medical and other carers to help raise awareness
of MPS.
The Society would be interested in your ideas for how to
promote MPS Awareness at our next MPS Awareness Day on 15 May 2008.
Strategic Planning for the Society for
the next 5 years
The Committee of Management and Board of the
Australian MPS Society are current working on a strategic plan for the
Society for the next 5 years. A planning day was held on 21
April 2007 and a further get together was held on 16 June.
Details of our strategic plan and reviewed Mission and Aims will be
posted in due course.
