Thalassemia Patients and Friends

Discussion Forums => Working Towards a Cure => Topic started by: Andy Battaglia on July 24, 2007, 10:12:14 PM



Title: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on July 24, 2007, 10:12:14 PM
Thalagen™: Gene Therapy Treatment for Thalassemia trials will begin in 2008. $3,000,000 will be needed to fund this in its first year. Help fund this by going to http://www.myspace.com/patgirondi
where you can purchase Pat Girondi's CD for $13. All $13 for each CD will go into the fund to finance these trials. Spread the word and ask people to buy the CD. I just bought mine.  :wink


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Manal on July 26, 2007, 09:02:38 AM
Andy i will too and will spread the word

Thanks Andy

Manal


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Canadian_Family on July 26, 2007, 11:18:53 AM
Hello,

I just ordered mine, I think it is best when odering to send comments to the musician (there is a place for it) for great cause, I did the same.

Thanks



Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on July 28, 2007, 01:10:21 AM
Pat Girondi's, Orphan's Soul CD arrived today and I just finished listening to it.  :smiley  I enjoyed the cool vocal jazz and was happy to think my thirteen bucks was going to the gene therapy trial fund. I pay a lot of taxes. A lot. What are the chances that $13 from those taxes will go to fund the trials? My guess is slim to none. This really needs to change. I'm paying thousands yearly to finance wars. Those are my tax dollars! If taxpayers were actually given a choice where the taxes went, would we really be spending so much money on endless war? This has to change folks. We have to demand better of the people we elect.

Check out the CD and tell your friends. It for a good cause and cheap enough for people to consider.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: rutuja2000 on September 18, 2007, 11:21:31 PM
hi
im rutuja's mom
andy sir i also want to order that cd for fund but im staying in india.pl tell me how should i order from india.thanx.also guide me if i want to go for gene transplant theropy in trial base .is there any chance to me if i go fot that.rutuja is beta thal major of 7.8yrs old,getting 99transfusion till now,and serrum ferritine is near about 1250 microg/gl.i was trying for BT but i had no donor at all.
but i really want to operate my daughter.i give desferal as wel as kelfer also.now she is in gud condition as doctor says.
if u want any center for stem cell trans.or CBT with unrelated donor then pl tell me.
i have 4/6 matched donor from Relience Life Sciences,Mumbai.
i hope u get my problem and do the needfull.
have a nice day.bye
reply soon.
i heard abt Singapore General hospital.but huide me in this regard.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: KHALIFA on September 19, 2007, 12:44:38 AM
HI
   Dear all i read all what is written and i really felt what do you feel ..don't forget am a father of    X-thalassemia child am really felt that do you want to know and find out one day there is new transplant for thalassemia PpL like what you discuse a bout GENE THARAPY  ...
   Dear all i don't want any body get down a bout what i said but every body know there is always A HOPE  also me i hope one day i hear somebody find out good treatment for thalassemia ...

   am personally ask PROFF. LUCARALLY (i like to call him FATHER OF THALASSEMIA)  a bout the gene tharapy last april when i was in italy to make rotin check up for ABDULWAHAB i ask him what a bout the gene tharapy .. he just close his computer and start to talk with me a bout it he told me they start to make reserch since 1975 and every five years they say we are going to find out the result after five years more...
     and the reserch still there is no good news ... also he replay he told me the only treatment for thalassemia availeble now is BMT or stem celles like what i did for my son .. 
       Dear Rutuja  mother i know what do you feel  because as i said am afather of X-thalassemia child who i made for him 2 operations one in 2003 and 2005 .
    first one was BMT and the seconde is STEAM CELLSE from the same donor who was is his sister .. i pray every day for those children who they suffering from thalassemia and i fight for them right her in my country ,,, but we still i don't know how much and how long to find out the prober result of the GENE THARAPY ...
        Dear RUtaje and Rutaja mother my heart with you and belive me i got a feeling you talk a bout me also and my son ...
     in the end i would like to say
                 DON'T LOOSE YOUR HOPE  
                                      khalifa
                                 state of kuwait
                        ONE FOR ALL &ALL FOR ONE 
         


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: rutuja2000 on September 22, 2007, 12:00:01 AM
gud morning
Mr.khalif
i really want 2 cure rutuja.but iam helpless.
b'coz i cannt found any donor for transplant.i just want 2 go for stem cell transplant.
pl.pl. help in this regard.
thanks for reply.
waiting 4 ur reply.
from where i will did the stem cell transplant.
can u send the addresses of that centers who are did that transplantation
bye.
god bless you


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Zaini on September 22, 2007, 05:47:15 AM
Hi Rutuja,

just a little note,that wether you go for a BMT or stem cell transplant,in both cases you'll need HLA matched donor for your kid,and transplantation is not an easy process.it's a big decision.

ZAINI.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on September 22, 2007, 12:57:43 PM
Hi Rutuja's mom,

BMT is both costly and risky. I think if you go to Singapore Dr Tan will advise you to continue with your child's current treatment since your daughter is doing well with her health. Deciding to do a BMT is a huge decision and you need to be aware of the dangers. I do not want to mention any names but a member of this group recently lost their child after a BMT and several of the Maldivian children who have undergone BMT's have also passed away. This is very sad and makes me think we need to work very hard to make sure parents are well aware of the risks before proceeding. Some do succeed as with Omega and Khalifa's children, but it is not an easy path to follow. I spoke to Dr Lucarelli at the Dubai thal conference in 2006 and he told me that Khalifa's son was very fortunate to have survived, as most patients with severe graft vs host disease do not survive.

If gene therapy does prove to be a cure it will be many years before it is widely available as long term studies will be needed before it is ever approved. However, once more trials are underway, the patients chosen as subjects, will be those in the best of health. At this time there is no process for applying to be a participant in trials but if early trials show some success the trials will be expanded to include more patients. As with many drugs, it may be that the only way to get gene therapy for many years to come, will be to be part of a trial.

I have emailed Pat Girondi to inquire about shipping his cds to Asia. I will update when I get a response.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on September 22, 2007, 01:47:51 PM
Pat Girondi replied that the cds can be sent internationally.

Quote
We can ship it.
Sent wirelessly via BlackBerry from T-Mobile.

http://cdbaby.com/cd/patg


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: KHALIFA on September 26, 2007, 10:59:38 AM
Hi Rutuja,

just a little note,that wether you go for a BMT or stem cell transplant,in both cases you'll need HLA matched donor for your kid,and transplantation is not an easy process.it's a big decision.

ZAINI.
           Dear some body answer you before i did ..
      you must have 100% match donor ...
                  khalifa


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on October 07, 2007, 09:52:14 PM
I received the following update from Pat Girondi on the current progress towards the gene therapy trials and also about the fund raising efforts. If anyone knows any investors please let them know about this. It is an exciting investment opportunity that may eventually lead to cures for thalassemia and other orphan diseases and disorders. Check out the link to see Pat's new video.

Quote
"Dear Andy,
 
The vector is being tested. If it is a good batch we will then commence the making of the batch for the patients.
 
June 15th looks to be a good starting date for the first patient.  After that we can expect to see hemoglobin expression by the end of summer. Then it's tinkering to get the most effective scheme.
 
We need all the help that we can get. We have just come out with a PPM with the hope of raising 5 million dollars.
 
I am coming out with my second album and have added a link to a song that will be used in a movie that will be at Cannes, Berlin, Venice and hopefully out by January.

Here is the link to the updated "It's Your Time:"
 
http://www.youtube.com/watch?v=7A8TCsnnChA
 
Quote
I appreciate all the support. Right now we could use investors. The units are 50 k a piece, they of course are highly speculative like all biotech. the kicker is that they come with the 'Orphan Drug Tax Credit' which if you do the math really is a reduced price.
 
It works like this,
 
50k invested
20k returned in the form of the ODTC
12k returned if the company fails
----------------------------
 
Real risk is about 18k. besides it's an incredible company.
 
Thanks again.
 
pat
"



Anyone know any big investors?



Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Sharmin on November 06, 2008, 12:00:50 AM
Andy,

Do you know how things are going with this project? 
Sometimes it seems like this can't happen fast enough - yet I know the obstacles that need to be overcome before the trials can even start.  I hope that a new government means a new, more liberal mentality and more support for causes like this.

Sharmin


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on November 06, 2008, 09:17:38 AM
Good timing Sharmin. Here is the latest from Pat Girondi.

Quote
There is a lot of mis-communication about the 'Thalagen' project. I guess this is normal and natural for a project that is so significant for so many. This is not my project. This is the project of every person who has suffered from thalassemia, living and deceased.
 
I am but an infinitely tiny toggle on this 'Hope for a Cure' ship.
 
The invention of 'Thalagen' is a guarantee of a cure for the 1,000,000 or so people affected by thalassemia in the world (notice I did not or avoid using the word thalassemic as we are not thalassemics but people affected by thalassemia).
 
Thalagen once inserted into the patients stem cells will make the 'beta globin gene'.
Thalagen has cured 5 generations of Thalassemic mice and created 'genetic expression' (tiny amounts) in two monkeys of the 'human beta globin gene'.
 
Our greatest challenge is called 'titre'. It basically means getting into enough of the patients cells with the right copy of titre.
 
The Italian Telethon has recently donated 500,000 to help this problem. Cooley's Anemia International of Ron Capano in New York has donated 250,000 towards this challenge. The Giambrone Foundation of Italy (National Foundation) has donated hundreds of thousands towards the project.
 
Presently there are people collaborating on this project from Singapore on the east to Los Angeles on the west. Michel Sadelain and his open honest way of approaching life has made it possible to assemble an 'All Star' team.
 
We have a dozen researchers in New York, Farid Boulad, Lila in Thessaloniki, Franco, Aurelio in Italy, doctors in Los Angeles, Washington and Singapore who are working on this project.
 
We see the shore!! We see the shore!!
 
I over the years have been contacted by hundreds of hopeful patients. Recently I have been contacted by one who is very sad and down. He is suspicious of the project's possibility to succeed. He is afraid that funding is insufficient and that we will sit here in the bay without ever docking.
 
I can only say to him and all of us that there are no guarantees in life.
 
My son Rocco is now 18. I have been working towards the goal of a 'Cure' for 16 years. October was my anniversary as he was diagnosed in October of '92'.
 
Literally millions have gone into this project. We have all participated. I have tried to shore the project up with a company and thanks to this effort we have seen significant support.
 
Investors in Bio-tech are rare and extraordinary people of vision, hope and willingness to put their resources where their heart is.
 
Thousands have now bought music from my 'myspace' site, Amazon, CD Baby, ITunes etc. In fact September saw over 7,000 dollars raised for our mission from the music alone with almost 2,000 actual purchases.
 
We are presently writing the IND. It should be done by February. As soon as the FDA gives us the go ahead, which I envision being April we will begin to make the 'Thalagen' needed for the first 5 patients. My son WILL NOT be in the first 5 patients.
 
The patients will be picked by Doctors Bouland and Tisdale at Sloan Kettering and at the NIH. We envision no problem in finding patients as the initial therapy will be very 'friendly' with little or no side effects.
 
The first patients should begin in the summer of '09'.
 
Our goal is 'Genetic Expression' of Thalagen, the beta globin gene.
 
If we achieve this it will be literally earth shattering as it will mean hope not only for us but for all people who suffer from 'Genetic Disease'.
 
Clinical Trials usually go in 3 phases, PHASE 1, 2 and 3.
 
Phase one is about 'doing no harm' to the patient.
Phase two is about 'seeing if it works'.
Phase three is about 'dosage'.
 
Please forgive me for my simple language. I was forced into the military at 16 by a judge in Chicago and I never received a high school diploma.
 
We believe that we will go from Phase one and then immediately into Phase three. We believe this because we are convinced that the first patients will see 'Gene Expression' of Thalagen the beta globin gene. This is great news.
 
People have asked about centers and cost. Well, initially the cost will be that of a Bone Marrow Transplant, since in essence it is an autologous BMT.
 
The centers will initially be in the US but then quickly spread out to Singapore, Italy and Greece.
 
We have helped and will continue to help anyone that we can. We recently assisted a Romanian woman and her son who was feared to have G6PD. Doctor Luzzato of Genova resolved the problem.
 
I will personally do what I can to help all patients who hope for the cure. I like to think of myself as a sensible man with the interest of my large family (Orphan Disease) at heart.
 
Rumors that we are out of cash (could always use some) to continue... Rumors that in France (where there was some sort of experimentation going on in no way affiliated with us) that things have failed... Rumors that the treatment will be given only to a select few to people on some remote island in the South Pacific... Rumors that this is all a 'get rich quick' money making scheme... are all that people, rumors.
 
I can not control what people think or say. (The Bush Regime tried and failed). Nor do I want to.
 
To these rumors, I say.
 
Funding
 
We need funding. I will continue to look under every rock with my management and investment team. We will accept donations and investments. If I have to hold a cup on the freeway, accept a sheik from  ABU DABI, as an investor who has 30 wives (be nice if he had a few extra for me) or rob a cheese store in Italy... WE WILL GET INTO PATIENTS. Case closed. I hope that this is clear.
 
France
 
For years I have heard of and about a project reportedly in France. I have known researchers who have worked on this project. I have known the CEO of the company that claims to support the project. It makes little sense for me to speak about things that are not crystal clear and open. This experiment was NOT done in a way that I would have done things. Yet all experience can be beneficial. By the way 'Michel Sadelain' is French.
 
 
Accessibility
 
When and IF the treatment is available, I will do my utmost as I believe everyone on the team will do to get it to as many people, wherever possible. If some sheik  has a child affected by thalassemia and some extra wives, I will also work with him. Realistically, not all is always possible. We are already collaborating with people in Los Angeles, Minnesota, Washington, Singapore etc... By the way, I've never been to an island in the South Pacific.
 
Get Rich Quick Scheme
 
According to the Wall Street Journal it takes 18 years and 800,000,000 USD average for a drug to get to market. Michel invented this project 19 years ago. If the project is successful, people who invested may see profit. God bless them. We couldn't have done it without them and their willingness to risk. By the way, I don't see anything quick about this. I personally will invest my life in 'Orphan Disease Research'. If any profit is made by anyone it will also profit research.
 
Bad news... My third CD, 'Orphan's Hope' will be out in the beginning of 2009.
 
This is all.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Sharmin on November 06, 2008, 09:36:03 AM
Andy,

Thanks for putting this up - this is after all what we all want and what we should be supporting. 

Sharmin


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Zaini on November 06, 2008, 12:06:54 PM
 :pray :pray :pray

Zaini.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Sharmin on January 09, 2009, 07:56:34 PM
Hi thalpals,

I have been sending messages to friends and family to begin buying CDs again - If we all do so it should make a big difference.  I am praying for this therapy to work.

Sharmin


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Manal on January 09, 2009, 08:06:32 PM
Thanks Sharmin, i will do the same too. Lets all pray for their success


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Sharmin on January 26, 2009, 08:37:04 PM
Hi Thalpals,

Something for us to look into:

Bill & Melinda Gates Foundation

http://www.gatesfoundation.org/Pages/home.aspx

Sharmin


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Manal on January 27, 2009, 07:45:13 PM
Thanks Sharmin, i will contact them ,hopei receive a reply

manal


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: ANI on May 06, 2009, 01:36:25 AM
When my Son was first diagnosed with Thal major, I was looking towards the gene therapy very eagerly and impatiently. I use to read all the news related to it and when I saw the web site of pat Girondi ,I was almost thrilled. In errantgene.com they have mentioned Thalgan as a product in pipe line and that the RAC has been approved by FDA in 2007.
But now standing in may 2009, it seems like a oassis n desert. More close you are more further you become. First trial. first dose, vector, lentiviral and what not.
Friends, I have not lost my faith but perhaps the force whch requires for driving the trial is somhow missig. People are concerned to extract the investement  from the limited patient base of Thalasemia.TRhey are more concerned about their ROI then serving the mankind. The funding from State government is insufficient. The demonstaration and road show from us is lacking. The profit factor more dominat then  Human care factor.
I think , if all of us want to see it as a success we shoukld not just wait for Mr Pat girondi CD to sell rather we should write to our state Giverment to arrange for fund amidst Economic meltdow. The priorities for research for thalassemia needs to be decided and worked upon.
Let all of us take a pledge to write at least 100 letter to all that Governement Organization, NGO's, Volunteering organization, Rich individual and who so ever matters.
If person like Bill and Melinda gates hear to us and person like Tiger wood supprt us, perhaps the day os success will not be far.
Lot of Regard to all of you who are directly or indirecly helping Thalassemics and not taking it as a burden.I am doing my part of job and will continue doing this, I promise.



Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: suk838 on May 06, 2009, 01:02:30 PM
really hope gene therapy will sucess and will be the cure for all thalassemia patient soon after.




Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Parin on September 02, 2009, 11:41:19 AM
GENE THERAPY TO MAKE HISTORY IN OCTOBER

Dear family member…

I leave for Pavia in the evening. It will be a historic trip.

Your dreams and prayers, your hard earned resources have made the trip possible.  A major disease, (thalassemia, my son Rocco and your son Rocco’s gift) will be treated genetically for the first time.

I will not rant about all of the success that has accompanied us though the halls of the FDA and the canals of science.  I would like however to thank you for your presence as comrades and mentors.

October is the 17th anniversary of Rocco’s diagnoses. October is the month when the first patient will leave for the US from Italy to be treated. I don’t know who he is but, for me, it is Rocco. The patient will go through the various phases and begin the therapy after these things are finished.

Your company has grown to 17 patents. We will next tackle Sickle Cell Anemia. Everything that we have learned until now will shorten the trip for Sickle Cell.

I will probably spend more and more time in the Mediterranean where there are a hundred thousand patients. I will build here as we have built in the US. As you know we have collaborating centers everywhere from LA to Singapore.

Our new president is paving the way for a business plan that will ensure that the Orphan Diseases are always a consideration and the life of EGT.

May the Orphan Dream be your dream.




Pat Girondi



Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on September 02, 2009, 12:03:12 PM
Pat,

Best wishes for success and a real cure.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Sharmin on September 02, 2009, 12:06:22 PM
Pat,

Best wishes that this will be successful for the patient who will be treated and for everyone else,

Sharmin


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Zaini on September 02, 2009, 12:21:58 PM
Pat,

Thanks for sharing this,may our dream of cure come true  :pray .

Zaini.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: suk838 on September 03, 2009, 12:27:59 PM
Good luck and all best, praying for the good news.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: nice friend on September 05, 2009, 10:58:56 PM
Beest Wishes & Regards :pray :pray


Umair


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Manal on September 15, 2009, 12:09:44 AM
So excited!!! Wish you all the luck, you have been working hard on this and definetly God will grant us a cure

manal

 :pray :pray :pray :pray :pray :pray :pray


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on January 01, 2010, 06:12:43 PM
2010 will be a very big year in the world of thalassemia. The group led by Dr Michael Sadelain is about to begin the gene therapy trials in humans and this should commence by springtime. By the end of this year, we should have a good idea if gene therapy will provide the long awaited cure for thalassemia. I believe that this will succeed and that the only thing that has held it back is lack of funding. As long as my government makes financial decisions that places paying for drones to launch missiles over the heads of many of my friends, who also await this cure, we cannot hope that this same government will EVER give the same priority to saving human lives as it does to destroying them, so we must find funding elsewhere. Our friend, Pat Girondi has dedicated much of his life to helping to fund a cure for orphan diseases and especially for the thalassemia that has touched his own son. Pat donates all proceeds from the sale of his CDs to the Orphan's Dream Foundation to help fund these gene therapy trials. If you like music and want to make a small donation, and in turn get some fine music, please go to this website and buy one or more of Pat's CDs. You will be helping to fund this incredible work that will eventually provide a cure for thalassemia.
http://www.cdbaby.com/cd/patg3#

(http://i71.photobucket.com/albums/i126/comicsetc/Misc/patg3.jpg)


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Dori on June 04, 2010, 03:01:42 PM
 :thumbs


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Zaini on June 05, 2010, 10:23:03 AM
As always Andy,you are spot on,i just i hope that i'll see the cure in my life time.

Zaini.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: 7assan on June 06, 2010, 10:05:18 AM
 what is new in gene therapy..................? ???


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on June 06, 2010, 10:17:42 AM
The US trials will be starting this autumn. Hopes are high, as the preliminary work has gone even better than expected. I will post regular updates as I receive them.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: 7assan on June 06, 2010, 10:25:34 AM
great news  and thnx andy for the updating me


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: aabha on June 06, 2010, 01:11:38 PM
GREAT NEWS!!hope we can have it in india real soon!
aabha


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Sharmin on June 06, 2010, 03:48:55 PM
I cannot wait for these trials to begin!  I think that these trials will lead to a lot of success in treating this disease.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Parin on June 02, 2011, 11:15:51 AM
Andy, is there any updates about the trial and all? OR anyone knows about these trial?

Thanks

AshutoshT


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: MikeD on January 20, 2013, 01:46:33 PM
Hi everyone- just curious what sort of fundraisers people from thsi board have held to help raise awareness and money to donate towards funding for a cure?  It would be nice to share some ideas.  We are in inital stages of learning more about thal and whats ahead but we have always been active in community contributions and would love to put something together for the upcoming summer possibly in our local area. 


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Andy Battaglia on January 20, 2013, 05:43:55 PM
You can also learn more about fundraising in the US at
http://www.thalassemia.org/index.php?option=com_content&view=section&layout=blog&id=12&Itemid=61


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Parin on November 18, 2014, 12:00:53 AM
Updates from Pat G

Dear Family member,
 
When my communications falter, it means that I am praying. My mother taught me the power of prayer; experience teaches me the power of man.
 
I am Catholic, Buddhist, Sikh, Hebrew, Atheist, and Muslim. I am all of these things as sure as love binds us.
 
We have treated 3 patients with the SKI-EGT collaborative vector. We used myelosuppression, not myeloablation. The difference is that one suppresses the bone marrow and the other kills it.
 
One of the 3 patient has reduced transfusions by 50%. This is amazing and I know that I (for Rocco) as well as many of you would stand on your head in line to reduce transfusions by 50%.
 
We’re obviously aiming for a cure. Unfortunately, the ship that was in port has been pushed back out to sea. I’m not really sure why this happened. There is no doubt that this project has attracted the attention of all sorts of people. There is no doubt that conflicting agendas are coming into play so I am following what my mother taught. When you don’t know what else to do, pray with all of your heart. I am asking you to do this for our project.
 
We are so close.
 
My love always,
 
pat girondi


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Sharmin on November 19, 2014, 12:21:20 AM
I am constantly praying for these trials to continue and for the patients to thrive.  This message has me worried.  I hope things will work out soon.


Title: Re: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials
Post by: Bostonian_04 on November 25, 2014, 03:28:53 PM
what does the message from Pat mean? I hope things go well and they succeed. prayers for them.