Hello peeps, thank you for accompanying us for the past 7 weeks, it has been a blast!! I'm sad to say that we have come to the end of our blog posts :((. But!! I hope you all had an enriching journey, you have certainly learnt about a new disease! :D Thank you for your support and if u still have any questions regarding Pompe disease you can still post your comments. Adieu~
Mei Ying & Rose
Sunday, January 4, 2015
Saturday, January 3, 2015
Movieeeeee
YOOOOOOOOO. Last day of term break today so I'm gonna share a MOVIEEEEE with you guys! YES, THERE'S AN ATUAL MOVIE ABOUT POMPE DISEASE, U GUYS EXCITED???
So, the title of the movie is called " Extraordinary Measures". It's a 2010 medical drama film starring Brenden Fraser (from the Mummy series), Harrison Ford (Indiana Jones!) and Keri Russell. This film is based on the true story of John and Aileen Crowley, whose children has Pompe disease and they form a biotechnology company to develop a drug to save the lives of their children.
Brenden Fraser & Keri Russell ass John & Aileen Crowley
John & Aileen Crowley with their children
Below is the movie trailer for Extraordinary Measures.
Thats all for today , bye guys, enjoy your last day of holiday!
Reference:
http://www.filmofilia.com/extraordinary-measures-review-12925/extraordinary-measures-08/
http://www.nbcnews.com/id/35199811/ns/us_news-giving/t/celebs-tout-charitable-extraordinary-measures/
Rose
So, the title of the movie is called " Extraordinary Measures". It's a 2010 medical drama film starring Brenden Fraser (from the Mummy series), Harrison Ford (Indiana Jones!) and Keri Russell. This film is based on the true story of John and Aileen Crowley, whose children has Pompe disease and they form a biotechnology company to develop a drug to save the lives of their children.
Brenden Fraser & Keri Russell ass John & Aileen Crowley
John & Aileen Crowley with their children
Below is the movie trailer for Extraordinary Measures.
Thats all for today , bye guys, enjoy your last day of holiday!
Reference:
http://www.filmofilia.com/extraordinary-measures-review-12925/extraordinary-measures-08/
http://www.nbcnews.com/id/35199811/ns/us_news-giving/t/celebs-tout-charitable-extraordinary-measures/
Rose
Friday, January 2, 2015
Campaigns and support for Pompe Disease
Over the years, Pompe Disease has been gaining awareness globally which is quite a feat considering that it is a rare disease. So today we will be looking at what makes this possible.
There have been campaigns that have been spreading around which promotes the awareness of Pompe. One such campaign which I have mentioned before in my previous post is the International Pompe Day. As said in the name, this is an INTERNATIONAL event which involves many countries. In support of International Pompe Day, a few countries have took the effort to raise the issue of Pompe to either the general public or the government with the intention of providing support to the local Pompe patients. Here are some example of the events that occur in some of the countries on International Pompe Day:
In Australia, Pompe disease is not reimbursed by the government. Hence, messages regarding this issue was actually sent to the Prime Minister of Health. Information of Pompe disease such as what the disease is about and how it affect patients were published in the Australian newspapers which brought the country's attention towards Pompe disease.
In the United Kingdom, a 15-year-old girl whose sister is suffering from Pompe disease, organised a marathon cum fundraising event for International Pompe Day and even shared her family's Pompe experience with the local media.
In Hong Kong, a video about an infant named Kei Kei, a local Hong Konger who was suffering from infantile-onset Pompe disease was aired on Hong Kong's TVB channel.
There are many other countries who did the same, the above are only some of the examples.
Other than campaigns to promote Pompe, there are also organizations set up to provide support to Pompe patients. One such organization is the Acid Maltase Deficiency Association (AMDA). Acid maltase defiency is another term used to call Pompe disease. AMDA was set up to promote Pompe to the public as well as to help in funding research which are working on Pompe disease.
Another example is www.pompe.com which is set up by Genzyme Corporation. This website serves as a platform for Pompe patients to get information and advice about the disease, provides tips to caretakers of Pompe patients and even provide information for healthcare professionals. The website also allows the general public to know more about the lives of Pompe patients by showing video interviews of the patients who talks about their experiences and feelings towards coping with the disease.
As you can see, the Pompe community seems to be a very close knit and supportive community <3. Hopefully they can continue to provide support for each other and spread awareness of the disease to improve the lives of the fellow patients! :)
References:
http://www.amda-pompe.org/
http://www.worldpompe.org/index.php/news/525-resounding-success-international-pompe-day-2014
http://www.pompe.com/
There have been campaigns that have been spreading around which promotes the awareness of Pompe. One such campaign which I have mentioned before in my previous post is the International Pompe Day. As said in the name, this is an INTERNATIONAL event which involves many countries. In support of International Pompe Day, a few countries have took the effort to raise the issue of Pompe to either the general public or the government with the intention of providing support to the local Pompe patients. Here are some example of the events that occur in some of the countries on International Pompe Day:
In Australia, Pompe disease is not reimbursed by the government. Hence, messages regarding this issue was actually sent to the Prime Minister of Health. Information of Pompe disease such as what the disease is about and how it affect patients were published in the Australian newspapers which brought the country's attention towards Pompe disease.
In the United Kingdom, a 15-year-old girl whose sister is suffering from Pompe disease, organised a marathon cum fundraising event for International Pompe Day and even shared her family's Pompe experience with the local media.
In Hong Kong, a video about an infant named Kei Kei, a local Hong Konger who was suffering from infantile-onset Pompe disease was aired on Hong Kong's TVB channel.
There are many other countries who did the same, the above are only some of the examples.
Other than campaigns to promote Pompe, there are also organizations set up to provide support to Pompe patients. One such organization is the Acid Maltase Deficiency Association (AMDA). Acid maltase defiency is another term used to call Pompe disease. AMDA was set up to promote Pompe to the public as well as to help in funding research which are working on Pompe disease.
Another example is www.pompe.com which is set up by Genzyme Corporation. This website serves as a platform for Pompe patients to get information and advice about the disease, provides tips to caretakers of Pompe patients and even provide information for healthcare professionals. The website also allows the general public to know more about the lives of Pompe patients by showing video interviews of the patients who talks about their experiences and feelings towards coping with the disease.
As you can see, the Pompe community seems to be a very close knit and supportive community <3. Hopefully they can continue to provide support for each other and spread awareness of the disease to improve the lives of the fellow patients! :)
References:
http://www.amda-pompe.org/
http://www.worldpompe.org/index.php/news/525-resounding-success-international-pompe-day-2014
http://www.pompe.com/
Some updates on Pompe Disease
Hello guys, welcome back! Since you all are already quite familiar with the disease we will not be posting anymore information on the mechanism of Pompe but instead we will be posting some extra information on Pompe. So today I will be giving some updates on Pompe disease :D
On 15 April 2014, the International Pompe Association (IPA) celebrated its first annual International Pompe Day. The purpose of this day is to raise awareness of the disease and the treatments, to encourage screening of the disease for infants and also for fundraising. There were a string of activities lined up for International Pompe Day which includes an art contest and a photo gallery. Individuals with Pompe disease and their family members also raised awareness of this day through the use of social media such as Facebook by changing their profile pictures to that of the International Pompe Day picture and they even encouraged their friends to do so! A commemorative book was also set up by the Acid Maltase Deficiency Association specially for this day which captures the events that took place with regards to International Pompe Day. So remember, every year on 15 April is International Pompe Day!! Don't forget to show your support!! :D
On 27 May 2014, BioMarin Pharmaceutical Inc. dosed their first patient with BMN 701 in the phase III INSPIRE trial for Pompe disease. Huh???? What are you talking about? Alright let me explain further. Basically, BMN 701 is a GILT-tagged Recombinant Human GAA which consist of an insulin like growth factor 2 fused with acid alpha glucosidase. In the phase III INSPIRE trial, an experiment is being caried out to assess if BMN 701 is beneficial for late-onset Pompe patients. Thus, on the day of 27 May 2014, the company that is involved in this trial had given the first patient the trial drug. The Pompe community will be looking forward to the results of this trial. If successful, Pompe patients will have another treatment available to them :D So lets hope for the best outcome for the trial!
Another great news for the Pompe community is that research has found out that a combination of cancer-treatment drugs can help in the treatment of Pompe disease. In the treatment of Pompe, the recombinant human GAA enzyme (myozyme) is injected into the child patient. However, some children will produce an immune response to the enzyme, preventing the enzyme from taking effect. As a result, the patients died. The good news is that now research has found out that a combination of cancer-treatment drugs actually suppress this immune response. This allows myozyme to take effect in patients which are producing the immune response and thus saving their lives! The results of this research has improved the lives of some children who suffer from the immune response, and hopefully more children will stand to benefit from it too in time to come.
There are a few more updates on Pompe which you can look up on the Internet if you are interested, but that's all I have for now. See you soon!! :))
References:
http://amdapompe.ehclients.com/downloads/news/BMN_701_First_Dose_Press_Release_FINAL_5-22-14.pdf
http://www.amda-pompe.org/index.php/main/news/amda_to_celebrate_first_international_pompe_day
http://www.news-medical.net/news/20120106/Combination-of-cancer-drugs-shows-promise-against-Pompe-disease.aspx
On 15 April 2014, the International Pompe Association (IPA) celebrated its first annual International Pompe Day. The purpose of this day is to raise awareness of the disease and the treatments, to encourage screening of the disease for infants and also for fundraising. There were a string of activities lined up for International Pompe Day which includes an art contest and a photo gallery. Individuals with Pompe disease and their family members also raised awareness of this day through the use of social media such as Facebook by changing their profile pictures to that of the International Pompe Day picture and they even encouraged their friends to do so! A commemorative book was also set up by the Acid Maltase Deficiency Association specially for this day which captures the events that took place with regards to International Pompe Day. So remember, every year on 15 April is International Pompe Day!! Don't forget to show your support!! :D
On 27 May 2014, BioMarin Pharmaceutical Inc. dosed their first patient with BMN 701 in the phase III INSPIRE trial for Pompe disease. Huh???? What are you talking about? Alright let me explain further. Basically, BMN 701 is a GILT-tagged Recombinant Human GAA which consist of an insulin like growth factor 2 fused with acid alpha glucosidase. In the phase III INSPIRE trial, an experiment is being caried out to assess if BMN 701 is beneficial for late-onset Pompe patients. Thus, on the day of 27 May 2014, the company that is involved in this trial had given the first patient the trial drug. The Pompe community will be looking forward to the results of this trial. If successful, Pompe patients will have another treatment available to them :D So lets hope for the best outcome for the trial!
Another great news for the Pompe community is that research has found out that a combination of cancer-treatment drugs can help in the treatment of Pompe disease. In the treatment of Pompe, the recombinant human GAA enzyme (myozyme) is injected into the child patient. However, some children will produce an immune response to the enzyme, preventing the enzyme from taking effect. As a result, the patients died. The good news is that now research has found out that a combination of cancer-treatment drugs actually suppress this immune response. This allows myozyme to take effect in patients which are producing the immune response and thus saving their lives! The results of this research has improved the lives of some children who suffer from the immune response, and hopefully more children will stand to benefit from it too in time to come.
There are a few more updates on Pompe which you can look up on the Internet if you are interested, but that's all I have for now. See you soon!! :))
References:
http://amdapompe.ehclients.com/downloads/news/BMN_701_First_Dose_Press_Release_FINAL_5-22-14.pdf
http://www.amda-pompe.org/index.php/main/news/amda_to_celebrate_first_international_pompe_day
http://www.news-medical.net/news/20120106/Combination-of-cancer-drugs-shows-promise-against-Pompe-disease.aspx
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