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Foundation Newsline and Updates Promising Therapies for Niemann-Pick Type C Disease
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| The 2010 Alex and Laura Vaughan Memorial Golf Event featured a silent auction and dinner in addition to golf. | |
The 8th Annual Alex and Laura Vaughan Memorial Golf Event, held on June 19, was a huge success, raising $22,000 for the fight against Niemann-Pick Disease. Thanks to the many participants, donors and supporters and to cooperation from the weather, a wonderful day was enjoyed by those in attendance, all while benefiting a worthy cause.
Visit the Recent Events page for more details, including a message from the Vaughan family, and a photo of the winning team of golfers.
[June 21, 2010 mem]
Are Stem Cells or Gene Replacement Viable Therapies for NPC?
April 13, 2010: In response to recent questions from NPC families, the NNPDF asked Dr. Dan Ory, Chair of the Foundation's Scientific Advisory Board, about the possibility of using either stem cells or gene replacement as therapies for Niemann-Pick Disease Type C.
Here is Dr. Ory's response:
I know of no evidence that gene replacement therapy is effective for NPC disease. There may be work going on in this area, but there is nothing promising on the horizon.
[ Re. the suggestion ]... that stem cell therapy is efficacious in the NPC1 mouse model... I am not aware of this. In fact, in my own lab I replicated the experiments done by labs claiming to show benefit from stem cell transplantation, only to find that the protocols actually worsened symptoms and shortened the life span of the mice. Any claim that with our current technology that stem cell transplantation is beneficial should be met with extreme skepticism.
[Apr 15, 2010 mem]
Ty's Story in 2010 Neuro Film Festival
Sponsored by American Academy of Neurology Foundation
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| Karen Quandt and Ty Quandt (NPC) |
Hi all,
Here is the video of Ty (Ty's Story) that is on YouTube as part of the 2010 Neuro Film Festival sponsored by the American Academy of Neurology Foundation (AANF). They are having a Neuro Film Festival at the convention in Toronto this Sunday. It is the largest convention of neurologists in the world.
So far Ty's video has been viewed 1,057 times! They have given the YouTube link to the neurologists attending or interested in this conference, so many of those views were by neurologists. They may show Ty's video at the conference on Sunday; I won't know for sure until they post information this weekend. Dr. Patterson will be at this conference and I sent him the video so he would know it is part of the Neuro Film Festival on YouTube and may be shown at the conference as well.
They said they will be doing the AANF Neuro Film Festival again next year. The convention is in Hawaii next year and the winners of the Neuro Film Festival get to attend the conference. Since we have so many families making videos, maybe we should let everyone know about this and have the families submit their videos if they so choose!
Karen Quandt, MN
Board Chair, NNPDF
Mom of Ty Quandt (NPC)
[Apr 9, 2010 mem]
8th Annual Alex and Laura Vaughan Memorial Golf Tournament
Stone Tree Golf Course
Owen Sound, Ontario
Saturday, June 19, 2010
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Alex Vaughan, NPC Laura Vaughan, NPC 4/18/88 - 12/23/92 1/16/95 -11/8/01 |
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Mark your calendar for the 8th Annual Alex and Laura Vaughan Memorial Golf Tournament on Saturday, June 19. This golf tournament will be 4-person teams, Best Ball format. The $100 per golfer fee incudes 18 holes of golf, power cart and dinner, along with a chance to win exciting prizes at designated holes.
Please read this special letter from CCNNPDF Chair Tammy Vaughan, mom to Alex and Laura.
Click here for the Entry Form and Sponsor/Donor Form.
[Mar 31, 2010 mem]
Health Canada Approves Zavesca for Niemann-Pick Type C
First Authorized Treatment in Canada
Health Canada recently announced its approval of the drug Zavesca (miglustat) as the first authorized treatment for neurological symptoms of Niemann-Pick Disease Type C (NPC). Zavesca is not a cure for NPC, but it has shown promise in treating symptoms related to NPC and in slowing the progression of the disease for some patients.
In addition to Canada, Zavesca has been approved for use in NPC in the European Union, South Korea, Brazil, Russia and Australia. (Currently, in the United States, Zavesca can only be prescribed "off-label" for use in NPC.)
Produced by Actelion Pharmaceuticals, Zavesca is also used for treatment of patients with Type 1 Gaucher Disease, another lysosomal storage disease.
To read the CCNNPDF's press release regarding Canada's approval of Zavesca, click here.
To read the press release from Actelion, click here. Actelion's press release in French
[Mar 24, 2010 mem]
Raising Funds and Awareness
School Shows Support for Cardinal Family and Others Affected by NPD
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| Damon Eli Cardinal (NPA) 12/26/06 - 4/16/08 |
Dylan and his teacher, Mrs. Lashmar, with some of the items collected for the raffle |
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| Alexis and Dylan Cardinal with the Raffle Items | Alexis and her beautiful poster |
As the two-year anniversary of the passing of little Damon Cardinal (NPA) approaches April 16, St. Teresa's School, where Damon's three eldest siblings attend, planned a balloon release to honor Damon's memory. Nine-year-old Dylan Cardinal's teacher, Mrs. Theresa Lashmar, also coordinated an awareness and fundraising event in memory of Damon.
Mrs. Lashmar's students had the opportunity to purchase raffle tickets for movie passes and popcorn donated by Cineplex Odeon Theatre, with proceeds benefiting the CCNNPDF. The class watched a video and made scrapbook pages in memory of Damon for Dylan to take home as a keepsake.
Mar 25 , 2010 mem]
World Rare Disease Day |
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One in 10 Canadians has been diagnosed with a rare disorder. Many others are affected or at risk but remain undiagnosed and unaware. Rare diseases are chronic, progressive, debilitating, disabling, severe and often life-threatening. Information is scarce and research is insufficient. People affected face challenges such as diagnosis delay, misdiagnosis, psychological burden and lack of practical support. Many rare disease patients are denied their right to the highest attainable standard of health and continue to advocate their need to overcome common obstacles. |
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The family of Kaiden St. Germain is participating in Rare Disease Day by creating and distributing a flyer and lapel ribbons to raise awareness at Kaiden's school and around the community. They are also hosting a jewelry party on March 6th, with a portion of the proceeds to be donated to CCNNPDF. Click here to see the flyer, and click here to watch their awareness video. |
Kaiden St. Germain | |
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Little Damon Eli Cardinal, lost to Niemann-Pick Disease Type A at the age of 16 months, lives on in the hearts of his family as they work to increase awareness and raise funds for the fight against Niemann-Pick Disease. This video of a news story about Damon and the Cardinal family has now been uploaded to YouTube, spreading Damon's story and educating others about the disease just in time for World Rare Disease Day on February 28. Damon's nine-year-old brother, Dylan , and his teacher, Mrs. Theresa Lashmar, are planning an awareness and fundraising event in memory of Damon (NPA). Mrs. Lashmar's students will have the opportunity to purchase raffle tickets for movie passes and popcorn donated by Cineplex Odeon Theatre, with proceeds to benefit the CCNNPDF. The class will also watch a video and make scrapbook pages in memory of Damon for Dylan to take home as a keepsake. St. Teresa's School, where Damon's three eldest siblings attend, is also planning a balloon release to honor Damon's memory as the two-year anniversary of his passing approaches April 16. Click here to learn more about World Rare Disease Day and how you can get involved. |
| Damon Cardinal (NPA) 12/26/06 - 4/16/08 |
[Feb 17, 2010 mem]
Life for Luke Gala
Riviera Parque Banquet & Convention Centre
Vaughan, Ontario
Saturday, March 6, 2010
6:00 p.m.
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Update: The 2010 Life for Luke Gala was a great success!
Videos from the event have been posted to YouTube: Life for Luke 2010 #1 Life for Luke 2010 #2
A link to local coverage of the gala: Snap Woodbridge's Life for Luke article
The 9th Annual Life for Luka Gala is scheduled for Saturday, March 6, at 6:00 p.m., at the Riviera Parque Banquet and Convention Centre, Vaughan, Ontario. This event, held in memory of Luke Liegghio (NPC), will benefit the Canadian Chapter of the NNPDF and the Hospital for Sick Children. Scrumptious foods from around the world, superb service, and outstanding entertainment make for a spectacular evening not to be missed.
Singing sensation Daniel Panetta and comedian Frank Spadone will headline the evening's entertainment. Door prizes and one-of-a-kind silent and live auction items help ensure no one goes home empty-handed from this very special event.
Order tickets now for this spectacular evening of food, professional entertainment, dancing and silent auction deals.
For complete information, please visit our Life for Luke page, or the Life for Luke Foundation's Web site.
[Feb 10, 2010 mem]
Winter 2009 Newsletter
The NNPDF Winter 2009 newsletter is now available, full of all the latest news and information. Click here to read the newsletter online. The CCNNPDF's special insert is also available here. Paper copies of the newsletter are in the mail to all our member families and friends.
[Dec 15, 2009]
NPD Family Support and Medical Conference
August 5 - 8, 2010
Toronto, Ontario
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The 18th Annual Niemann-Pick Disease Family Support and Medical Conference will be hosted by the Canadian Chapter of the NNPDF (CCNNPDF) in the beautiful city of Toronto! The conference will be held August 5 - 8, 2010. For more details, please visit this page.
[Dec 2, 2009 mem]
Scentsy Fundraiser for Niemann-Pick Disease Research
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| Damon Cardinal (NPA) 12/26/06 - 4/16/08 |
Escential Consultant Janna Henke is hosting a "Scentsy Fundraiser for Niemann-Pick Disease Research" in memory of Damon Cardinal, son of Elsa and Delainy Cardinal of Edmonton, Alberta, Canada.
Scentsy offers unique wickless candles and specially designed ceramic warmers, using the heat of a light bulb rather than an open flame, to slowly melt the wax and release aromatic scents.
Elsa writes: "The products are amazing, and will make wonderful Christmas gifts for yourself or anyone you know. Twenty percent of the total product sales are being donated to CCNNPDF [the Canadian Chapter of the National Niemann-Pick Disease Foundation]!"
Scentsy products can be shipped and orders may be placed online by visiting Janna's Scentsy page. Thank you, Janna and Elsa!
[Dec 4, 2009 mem]
YouTube Video of "Our Kaiden" Raises Awareness of NPC
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| Kaiden St. Germain, age 3 |
Sharla St. Germain, mom to Kaiden, recently posted to YouTube a very educational video regarding Niemann-Pick Disease Type C. The video, set to music, features darling three-year-old Kaiden and his sister and brother and parents, as well as information about the disease and its diagnosis.
Click the link to view the video: http://www.youtube.com/watch?v=piJJJ9aB_Oc
Thank you, Sharla, for your continuing efforts to raise awareness of Niemann-Pick Disease!
[Nov 20, 2009 mem]
H1N1 Flu and Seasonal Flu Information and Resources
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Seasonal flu and the H1N1 flu are concerns on most everyone's minds. Families with members whose health is compromised by chronic illness should take special care to avoid contracting the flu. Sick Kids Hospital in Toronto provided us with this information for families with regard to H1N1 flu and vaccination: Q and A for Parents of Children with Chronic Medical Conditions |
Please visit the NNPDF's H1N1 and Seasonal Flu Information page for additional resources and official information about avoiding the flu, and for dealing with it should a member of your family contract it despite precautions. |
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[Nov 4, 2009 mem]
Kaiden St. Germain featured in Medicine Hat News
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| Kaiden St. Germain (NPC) |
Three-and-a-half-year-old Kaiden St. Germain's journey to a diagnosis of Niemann-Pick Type C was featured in a recent article in the Medicine Hat News. Click here and here to see the article, written by Tenille Tellman.
[Oct 28, 2009 mem]
October is National Niemann-Pick Disease Awareness Month!
Raising Awareness and Funding for Research
October has begun, the month designated as National Niemann-Pick Disease Awareness Month. This is the time when we make an extra push to educate the public, extended family and friends about Niemann-Pick Disease, the devastation it causes families, and the need for essential research.
For October 2009, the 8th Annual Niemann-Pick Disease Awareness Month, we are pulling out all the stops to raise awareness and funding which is so critically needed to ensure that one day, NPD will no longer be such a dire diagnosis. Check out the October Awareness page and the Current Fundraising Events page on the NNPDF Web site to see all that families around Canada and the U.S. are doing to raise awareness and funds.
The NNPDF is busily supporting these families in their efforts, and we would like to encourage everyone to consider doing something in their local community to raise awareness. This can be as simple as sending a press release (which we can help you write or edit), or asking your local city officials to proclaim October as NPD Awareness Month (we have samples and can help you do this, as well). If you are more ambitious, it's not too late to launch a fundraising event. Check out the pages linked above for some great ideas, from the most simple to the more complex.
Thank you for all you are doing this month, and all year through, to help us raise awareness and funds for our all-important Quest for a Cure!
[Oct 7, 2009 mem]
NNPDF Representatives to Attend First International Conference on Niemann-Pick Disease
The first International Conference on Niemann-Pick Disease is being held October 8 - 11, in Scotland, co-sponsored by the NNPDF and the NPD Group (UK). The purpose of this conference is to enhance communication and representation among the organizations supporting those with NPD around the world.
Collaboration will help maximize efforts and avoid duplication of work, in good stewardship of hard-earned resources. This conference marks an important step in global collaboration and cooperation in the fight against NPD.
The NNPDF and CC-NNPDF will be represented by: Barb Vorpahl, NNPDF Board Chair; Karen Quandt, NNPDF Vice Chair; Tammy Vaughan, NNPDF Secretary and CC-NNPDF Chair; Lucy Liegghio, CC-NNPDF Vice Chair; and Nadine Hill, NNPDF Director of Family Services.
Thanks to Actelion for financial support!
[Oct 7, 2009 mem]
Minde-Redcrow Family and Friends Letter, Jewelry Party, etc.
Tessa Minde and Warren Redcrow of Hobbema, Alberta, Canada, parents of 15-month-old Tehyez Minde (NPA), are sending out Family and Friends letters during October to raise awareness and funds to help fight NPD.
A friend of Tessa's also hosted a 5th Avenue Jewelry Party at her workplace, and as the hostess, she was entitled to 15% of the profit, which she donated in honor of Tehyez.
The school where Tessa works is planning to do a fundraiser, as well, and Tessa is in contact with a local newspaper about doing a feature story on Tehyez and Niemann-Pick Disease.
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Tehyez Minde (NPA) |
[Oct 7, 2009 mem]
Cardinal Family and Friends Letter, Mayor's Proclamation, and Persevere Items Sales
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Damon Cardinal (NPA) 12/26/06 - 4/16/08 |
Elsa and Delainy Cardinal of Alberta, Canada, will again mark October Awareness Month with a Family and Friends Letter in memory of their son, Damon Eli Cardinal. Sixteen-month-old Damon lost his battle with Niemann-Pick Type A in 2008.
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| Renee and Alexis, raising money and awareness for NPD |
Alexis Cardinal, sister to Damon, and her friend, Renee, set out to raise $100 in memory of Damon, going door to door handing out brochures, sharing about Damon, and collecting donations from the sale of Persevere water bottles and wristbands.
Proud mom, Elsa Cardinal writes, "Although they were discouraged at first with a lot of 'no's' they PERSEVERED and raised over $40 within an hour. Both girls were smiling proudly and since then another friend has joined them to try and sell the last items they have in their bag. Alexis and my 2 oldest boys are also looking forward to handing the letters I ordered to their classmates on Friday. Alexis plans to take water bottles to school as well and ask for donations from her classmates to help her fulfill her goal."
Further, the Cardinals, together with Tessa Minde and Warren Redcrow (parents of 15-month-old Tehyez, NPA) requested Edmonton, Alberta Mayor Stephen Mandel, to proclaim October as Niemann-Pick Disease Awareness Month in the city. See the proclamation here.
[Sept 30, 2009 mem]
St. Germain Family and Friends Letter
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Kaiden St. Germain |
Sharla St. Germain is sending out a Friends and Family Letter in honor of her son, Kaiden St. Germain. Kaiden is still undergoing testing in what has been a difficult diagnostic process.
[Sept 25, 2009 mem]
Hudson - Cardinal Wedding
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| The Cardinal Family |
Elsa and Delainy Cardinal |
Damon Eli Cardinal (NPA) 12/26/06 - 4/16/08 |
Elsa Hudson and Delainy Cardinal recently married, with wedding photos taken by Jenny Alston of Imajen Portrait Art. Jenny donated her photography fee to the Canadian Chapter of the NNPDF in memory of the Cardinal's son, Damon Cardinal. Damon passed away at the age of 16 months, in April of 2008, from Niemann-Pick Disease Type A.
Congratulations to the Cardinal family, and Thank You to Jenny Alston for supporting NPD research and support services for families like the Cardinals!
[Sept 23, 2009 mem]
2010 NNPDF Family Support and Medical Conference to be held in Toronto
Hello NNPDF Families and Friends,
It is official ~ the 18th Annual NNPDF Family Support and Medical Conference to be hosted by our good friends to the north,
the Canadian Chapter of the NNPDF, will be held in the beautiful city of Toronto, Ontario, Canada.
To see and learn more about all that this wonderful destination has to offer, please visit the "See Toronto Now" web site:
http://www.seetorontonow.com/
The families of Tammy & Alan Vaughan and Lucy & Enio Liegghio, of the CC ~ NNPDF, have graciously stepped forward
to co-host this event in memory of their precious children: Alex Vaughan (NPC ~ 04/18/88-12/23/92), Laura
Vaughan (NPC ~ 1/16/95-11/8/01), and Luke Liegghio (NPC ~ 5/5/97-6/10/05).
The Vaughans & Liegghios will work hand-in-hand with the Family Conference Committee of the NNPDF to develop a
meeting that will best meet the interests and needs of our family membership. The planning for this event has begun in
earnest and we will forward information on a firm date and location announcement for the conference as soon as possible.
For those who like to get their family calendars in line early on ~ please pencil in late July 2010 and/or early August 2010
as the most likely date options for this event.
We wanted to share the excitement over this Family Conference location site choice with all of our families and members
so that you too can join in the fun and begin planning your family trip for next summer!
In the interim, you may wish to visit our NNPDF Web site to view updates from the wonderfully successful 2009 17th Annual
NNPDF Family Support and Medical Conference which was just held in Seattle, Washington, hosted by the family of Karen &
Gene Quandt, in honor of their son, Ty (Age 12, NPC). http://www.nnpdf.org/familyservices_03.html
This link includes two fun slide-show recaps and a link to the memorial video from this event. We are also working on
fine-tuning speaker recaps summaries and PowerPoint presentations so that we can post this information for all of our
members who weren't able to travel to Seattle and attend the conference in person.
Great thanks go out to the families who continue to step-up and assist the foundation with the planning of the annual
conferences ~ it is so greatly appreciated.
Kind Regards,
Nadine
[Sept 10, 2009 mem]
"In the News"
Updates on OrphaNews
Newsletter of the Rare Diseases Task Force
Visit OrphaNews Europe to read the following updates:
National and International Policy Developments: A Canadian province adopts rare disease drug evaluation programme.
Ethical, Legal and Social Issues: An international expert group reiterates the need to adhere to guidelines for stem cell clinical applications.
[July 13, 2009 mem]
7th Annual Alex and Laura Vaughan Memorial Golf Tournament
Saturday, June 20, 2009
Stone Tree Golf Club, Owen Sound, Ontario, Canada
11:00 a.m.
Alex Vaughan, NPC Laura Vaughan, NPC
4/18/88 - 12/23/92 1/16/95 -11/8/01
This golf tournament will be 4-person teams, Best Ball format. The $100 fee incudes 18 holes of golf, power cart and dinner, along with a chance to win exciting prizes. Hole sponsors, raffle prize donations and monetary donations are also being solicited in support of this event. Proceeds will benefit the Canadian Chapter of the National Niemann-Pick Disease Foundation.
Click here for more information, including a registration form and sponsorship/donation form.
A note from Tammy Vaughan, mother of Alex and Laura, and Chair of the Canadian Chapter of the NNPDF, about the 2009 Vaughan Memorial Golf Tournament:
Thanks to our friends, family, and the generous businesses throughout Canada and the US, we will be contributing another $24,000 to research to find a treatment and cure for Niemann-Pick Disease. This brings our 7 year total to over $159,000!
Every year I am literally overwhelmed by the amazing support that we receive – you guys are simply the best – what more can we say but THANK YOU, THANK YOU, THANK YOU.
The rain poured right up until tee-off time, but our little angels were looking out for us and the rain stopped at 10:55 am - leaving us dry for the remainder of the day. From what I have heard rain surrounded the area so I think that Stone Tree actually got some special treatment!
Once again, thank you from the bottom of hearts for your support and your generous contributions to this cause that is so important to us.
See you next year on the 19th of June!
Allan, Tammy and Michael
Look to this day
For yesterday is but a dream,
And tomorrow merely a vision.
But today, well lived,
Makes every yesterday a dream of happiness
And every tomorrow a vision of hope.
Look well, therefore, to this day
[June 22, 2009 mem]
Save the Dates!
NNPDF 17th Annual Family Support and Medical Conference
July 30 - Aug 2, 2009
Seattle,Washington
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The NNPDF's 17th Annual Family Support and Medical Conference is being planned for July 30 - August 2, 2009. The conference will be held in Seattle, Washington, at the Doubletree Guest Suites. We recommend you make your hotel reservations as soon as possible! Use the link to book your room as part of the NNPDF reserved block. We hope you will join us to learn the latest in research and treatments, and to build your support network and friendships with other families affected by Niemann-Pick Disease! Member families -- conference registration packets have been mailed out. You can also view the packet at the following link, along with airline info, etc. Please return your conference registrations by July 1st. |
[May 19, 2009 mem]
Update from Tammy Vaughan, CCNNPDF Chair, March 2009
CTV Calgary Television News Coverage
Who Inspires U?
Mariarosa Martino and her friend, Marchella Mafrica, garnered television coverage via a Calgary news program. Mariarosa has NPD Type B and is a strong supporter of the Canadian Chapter of the National Niemann-Pick Disease Foundation. Click the link below to read the story and to see the news video.
(The link to the video is in the center of the page titled: “Sage Pullen talks to a young woman with a rare disease.”)
Also, Mariarosa’s friend Marchella entered a video contest, "Who Inspires U?" Her video about their lifelong friendship made the top 20!
Thank you to these two young women who are certainly an inspiration to us all in their ongoing friendship and support of one another.
[May 19, 2009 mem]
Latest Research Highlights from the National Niemann-Pick Disease Foundation Research Committee Chair, Susan Green, and our Scientific Advisory Board Chair, Dr. Dan Ory, MD.
The latest research from the NNPDF ~ First Quarter 2009
[March 9, 2009 nmh]
National Niemann-Pick Disease Foundation
Peter G. Pentchev Research Fellowship
Request for Applications Issued
The National Niemann-Pick Disease Foundation (NNPDF) invites applications for postdoctoral research fellowships examining the biology of Niemann-Pick Type C (NPC) disease, a lethal neurodegenerative disease for which there are no effective therapies.
M.D., M.D.-Ph.D., and Ph. D. postdoctoral fellows are eligible to apply for funding to improve our understanding of the biology and pathogenesis of NPC disease. Preference will be given to research projects developing new therapies for NPC and identifying biomarkers of disease activity for diagnosis and clinical trials.
The fellowships provide support of $50,000 per annum for two years and may be renewable based on performance. Applicants must be currently associated with a recognized laboratory.
Applications are due May 1, 2009, and should be submitted by email to the Research Committee Chair.
Click here for application procedures.
Applicants will be informed of the funding decision by August 15, 2009, via email.
Two fellowships will be awarded to begin September 1, 2009.
For further information please contact the Research Committee Chair.
Click here for a printable flyer regarding the Peter G. Pentchev Research Fellowships.
[March 3, 2009 mem]
Niemann-Pick Disease in the National News
Dillon Papier (NPC)
Niemann-Pick Disease was the subject of a CBS News segment which aired in the United States on March 1st, featuring the Papier family of Frederick, Maryland.
Mark and Darrile Papier are the parents of Dillon, age 6, who is affected by Niemann-Pick Type C. The Papiers are members of the NNPDF, and Darrile recently joined the Foundation’s Board of Directors.
To view the segment, visit: http://www.cbsnews.com/video/watch/?id=4836925n&tag=main_home_MediaBox.
Please view the link, post your comments, and forward the link to your family and friends. Our goal is to make Dillon’s segment the most-viewed story on the CBS Web site.
If enough interest is shown in the segment, perhaps CBS will do a more in-depth story and help raise awareness of our quest for a cure!
[March 3, 2009 mem]
Expression of Hope is a global program featuring works of art by the community touched by lysosomal storage disorders (LSDs). Our vision is that those who experience this art will be inspired and moved by the powerful expressions of human spirit which the pieces will reveal. People living with LSDs, or caregivers around the world, are invited to submit artwork which shares their feelings of hope and expresses their innermost essence.
www.expressionofhope.com
022409 ~ nmh
Regions of Canada are adopting strategies for reimbursing medicinal products for rare diseases
The province of Alberta, Canada, last month announced a programme for rare disease pharmaceutical products. The Alberta Rare Diseases Drug Program is a new initiative that will reimburse Alberta residents for their rare disease treatments. The scheme requires users to pay premiums and “make co-payments consistent with their government-sponsored drug coverage”. The diseases currently eligible for coverage include Gaucher disease, Fabry Disease, MPS-I (Hurler/Hurler Scheie), Hunter syndrome and Pompe disease – all of which have treatments costing between C$250,000 - C$1million (€156,440 - €625,600) per year. The programme is expected to get underway in April 2009.
020109 ~ nmh
The Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the Canadian Chapter of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the CC ~ NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet.