Tag Archives: Amicus Therapeutics

From Extraordinary Leaders Come Extraordinary Measures

Bookmark and Share     In a few days a dramatic motion picture that could shape politics in one state will be coming to a theatre near you. I have not seen it yet, so it would be premature to declare it to be a cinematic tour de force but I do know it will be inspirational. I also know that it may finally launch the rise of a political force to reckon with from New Jersey.

 The movie, “Extraordinary Measures”, stars Harrison Ford and Brendan Fraser and it is based on the very real plight of John and Aileen Crowley who raced time and battled against all odds.

 John and Aileen were handed their greatest challenge when their children, Megan and Patrick were both born with a fatal neuromuscular disorder called Pompe disease.

Determined to change the deadly fate that doctors predicted, John and Aileen sought the care and treatment that could keep Patrick and Megan alive. But the disorder was so rare that few if any pharmaceutical entities attempted to develop effective treatments  for Pompe.  The disease was so rare that it lacked any widespread study. With so relatively few inflicted with Pompe, there was little if any profit motive for either the medical, scientific or pharmaceutical fields to devote much time, manpower and resources to it. Such a rare disease received barely even a fraction of the attention that more common health problems such as cancer receive.

The unimaginable situation that the Crowleys were dealt, compelled them to settle in Princeton, New Jersey so that they could be nearest to where some of the only doctors who had any experience in Pompe disease were located. But even in the hands of the most experienced experts of the disorder, John and Aileen were consistently forced to face the bleak diagnosis originally prescribed to their children.

Determined, John subsequently took up employment with Bristol-Meyers Squibb in the hope of fostering efforts to expedite a cure or treatment for the disease that was killing little Patrick and Megan.

After two years, with little progress and many roadblocks, he left Bristol-Meyers to break through the barriers and embark on a new venture to increase the pace of research into Pompe and the development of a cure. It was a race against time that was slowly taking the lives of his children.

Crowley joined forces with dedicated professionals and became the CEO of a biotech company called Novazyme. Eventually Novazyme merged with the world’s largest biotech company, Genzyme Corp., and soon after that the combined efforts of the two, brought about by John Crowley, expedited the creation of an enzyme replacement therapy for Pompe disease that is keeping the Crowley kids alive today.

Getting to that point was not easy and there were a great many trials and tribulations, but John Crowley, his wife, and the team he assembled, never gave up. The story is the stuff that books are written about. And before the movie was made about it, a book about the Crowley’s was written by a Pulitzer prize winning author.  It is called “The Cure: How a Father Raised $100 Million – And Bucked the Medical Establishment – In a Quest to Save His Children (ISBN 978-0060734398).

The experience now keeps Crowley hard at work as President and CEO of Cranbury, New Jersey based, Amicus Therapeutics. He believes that increased awareness about rare diseases, in general, is of great importance.

According to Crowley:

“There are 7000 rare diseases that collectively affect over 30 million people. That is more than everyone who suffers from all cancers and AIDS combined. So taken together these diseases are not rare at all – in fact they are remarkably prevalent. While the symptoms and severity of these rare diseases may vary, there are commonalities with regards to a framework of development for novel therapies that can and should be applied to all these disorders.”

Unfortunately, success stories like that of the Crowley family are far and few between. The uniqueness of their plight is one reason why it is so worthy of telling. The extraordinary measures that John Crowley and his wife took and the extraordinary feat of overcoming almost insurmountable odds to achieve a miracle of survival can only be accomplished by extraordinary people. Admittedl, few can be more motivated than a parent fighting for their children’s survival, but few have the faith, innovation and leadership required to make the impossible possible. Such qualities are what true leaders are made of. The type of leaders our nation needs more of. Leader’s who understand that personal freedom, can do more than people shackled by government burdens and blocked by government bureaucracies.

Perhaps that is why John Crowley is a Republican. He is not someone who will allow his fate to be written for him. He is determined to write his own. He is not someone content with being restricted by government but happier to get government out of the way.

It is also perhaps why, in the last few election cycles, John Crowley has been seen as a possible candidate for elected office in New Jersey. In 2008, there were some who hoped that Crowley would seek the US Senate seat currently occupied by the long serving, lackluster Frank Lautenberg.  Crowley failed to enter the race.

Later that year and in the early part of 2009, there was some speculation that John Crowley might run for Governor of New Jersey. Crowley declined to enter that race too.

I am one of those who held out hope for his candidacy.

Crowley is not a typical politician. He is a doer. His children’s lives are a testimony to that fact. He is not a go-along-to-get-a-long type. He is a man who gets what needs to be done, done. The type of person New Jersey and our nation needs in government, and his record is one of accomplishment, education and dedication.

Before his children were born, John was schooled for three semesters at the U.S. Naval Academy, graduated with degrees in foreign service from Georgetown University and went on to obtain his J.D from Notre Dame Law School before working as a litigation associate in the health care industry. He subsequently furthered his education and received an M.B.A from Harvard Business School. From there he went on to work for a San Francisco based management consulting firm.

Now, at 42, in addition to being a proud parent of three, devoted husband, as well as President and CEO of Amicus Therapeutics, Crowley is an intelligence officer in the U.S. Naval Reserve. He is also not shy about his political beliefs. As part of a political action committee called “Building the New Majority”, John assists a grassroots network that helps launch the candidacies of good Republicans on the local, county and statewide levels.

 Whether or not John Crowley will himself ever be one of those candidates is still a question mark. What the future holds is anybody’s guess. At least anybody who is not John Crowley. I have a feeling he knows what he intends to do.

Despite the recent election of a Republican Governor, New Jersey has few figures who are in any great position to win a statewide office in a state that leans so heavily to the left. But John Crowley would be one the few people who could be a strong candidate and who also has experience with uphill battles. The opportunity for statewide office will not come until 2012 when Democrat Senator Bob Menendez will be up for reelection. For that seat, there has recently been some speculation about financial journalist and anchorman Lou Dobbs running for the against Menendez, but if I had my druthers, the next Republican nominee for US Senate would be John Crowley.

Aside from knowing how to run a successful business that saves lives and aside from his having a track record of accomplishment that few can match, few candidates for elected office have a major motion picture made about them. That kind of introduction to the public can not be matched by any 30 second campaign commercial that airs during Desperate Housewives, when you get up to relieve yourself.

So I am looking forward to catching Extraordinary Measures   when it comes out on January 22nd .

Aside from promising to be one of those entertaining, feel good flicks, I look forward to seeing what I believe will be the launch of a new political rising star.

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“TOO MUCH CORRUPTION BECAUSE THERE’S TOO MUCH GOVENMENT”

antgovcoruptPOLITICS 24/7 has often stated that not only is state government too big but that there are too many governments in the state.

Based on the relatively small size of New Jersey, why there is a need for more than 650 municipal governments, is beyond me. Drive down any road in New Jersey and you will find yourself entering a new and different town every 3 or 4 minutes.

As stated here in the past, the proliferation of governments in this state is actually a major part of the problems that we face in the state.

The affordability crisis we are enduring is in large part due to the costs of operating all these governments. It costs a lot of money to operate and employ all these fire departments, police departments, borough halls, staffs, permit departments, and so on and so on.

It also creates an atmosphere ripe with the opportunity for corruption. Between the patronage, construction and service contracts, the ability to “spread the wealth around” runs rampant. But the wealth that is spread around is that of the taxpayers. While their wealth is taken away from them, those in charge of spreading it are doing so among their own friends, families, mistresses and fellow power brokers.

The existing arrangement has helped to make government one of the largest employers in the state. It has also helped to define New Jersey as one of the most state corrupt states in the Union, a title often in dispute with Louisiana but recently surpassed only by Illinois.

The arrest of Illinois’ Democrat Governor, Rod Blagojevich, for trying to sell President-Elect Obama’s newly available senate seat, along with other sleazy intentions, has helped take the title away from us. But we are still among the three most corrupt states at the top of that list.

Bridge commissioners, state contractors, council members, freeholders, county executives, judicial officials, cabinet members, party chairmen, state senators, assembly members and more have all been getting arrested, indicted, and sentenced in astonishing numbers.

Just today a former assembly candidate pleaded guilty in the same bribery scandal that took out his opponent for that same assembly seat.

For the past seven years, potential Republican candidate for Governor, former U.S. Attorney Chris Christie, has largely been responsible for exposing and successfully prosecuting the government corruption that has caused much of the public to lose their faith in public servants.

Having first hand experience with combating corruption, Christie knows about what he speaks of and today, speaking before an event sponsored by the New Jersey Business and Industry Association and Cherry Hill Regional Chamber of Commerce, the former crime buster stated that the reason for all the corruption in New Jersey was because “we just have too much government.”

Now that what we at POLITICS 24/7 have known all along has been confirmed by an expert in the area, what do we do about?

Well first of all, New Jersey needs to embark on the initiatives of others like Senator Joe Kyrillos of Monmouth County.

Senator Kyrillos has been pushing for consolidation. The type of consolidation which would make some of New Jersey’s less populous towns merge with larger neighboring towns.

This measure was actually proposed in legislation first sponsored by a former assemblyman, Republican Michael Arnone.

In the late ‘90’s, Assemblyman Arnone saw the need to curtail the spreading of governments in New Jersey the same way that we try to prevent the spreading of the flu.

Like a disease, the inordinate number of governments, along with their increasing size scope and staffs, have infected the state with a governing class that survives by doling out plumb, patronage positions and entering into corrupt contracts filled with kickbacks.

Does consolidation solve our problem? Nope, it sure doesn’t. Greed and other less attractive qualities that are sometimes a part of human nature will always exist. However, with less governments available for corruption to breed in, the less corruption will be born.

For me, Chris Christie’s remark is promising.

If he truly believes what he said, it may bode well for his possible candidacy. Small government conservatives will certainly appreciate the direction that his comment would take us in. Now if he can only expand on that remark.

If he does run for the Republican gubernatorial nomination, will he help to do more than offer lip service to state consolidation? Will he provide the initiatives of Senator Kyrillos with the type of support that is needed to influence liberal lawmakers to pass such measures?

Whether Christie runs or not, we need more leaders who are willing to solve our problems by acknowledging what the problems are and in New Jersey the problem is government.

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In international news, 60% of the people in the country of Jordan say they find Americans to be rude. Actually, that’s not so bad, when you consider 60% of people in other Middle Eastern countries think we’re Satan. …We’re moving up!

-Jay Leno

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WHY CAN’T FORMER U.S. ATTORNEY CHRIS CHRISTIE REACH A VERDICT ?

U.S. Attorney Chris Christie officially leaves office today

Chris Christie Was Sure Of Himself In The Past, So Why Does He Doubt Himself Now?


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Strategists behind the candidacy of former Bogota, N.J.  Mayor Steve Lonegan state that Chris Christie won’t be running for Governor.

One of Lonegan’s chief operatives claims that recent remarks by Christie, and others in his camp, are more indicative of a man who is not eager to run for the state’s top job and who is likely to forgo doing so.

Truthfully, I don’t put much weight in this claim.   However, the claim makes for a good strategy that helps to create an air of questions and doubt around Christie.

Nonetheless, the fact that it comes from a group of people who find Chris Christie to be their toughest opponent for the Republican gubernatorial nomination and their greatest obstacle to getting that nomination, makes this charge a little suspicious. It could very well be wishful thinking.

True or not,  Chris Christie’s declaring that he will will not make an announcement regarding his candidacy for another four to six weeks, is disheartening.

After the G.O.P. lost the presidential elections on November 4th, it was pretty clear that the newly elected President, a Democrat, would surely request Chris Christie, a Republican, to resign from the U.S. Attorney’s office .

Being aware of that, Christie gave notice and submitted his own resignation that took effect on December 1st.

On top of that is the fact that we saw the results of the presidential election coming for many weeks before Election Day. So what I am getting is this………….We all knew, for quite some time, that Chris Christie would be available for a new job soon. So we all anticipated that he would start submitting his resume to perspective employers relatively soon and we expected that those perspective employers would be us, the voters, and the position we would be interviewing him for was Governor of New Jersey.

We knew this and he knew this.

So what’s with Christie’s hesitation to declare his candidacy?

We hear that he wants to explore his opportunities and see where things are at.

But truth be told, such ambiguity from Christie is hogwash.

When one considers all the time he has had to make the decision to run for governor or not, one only conclude that he is playing some kind of game.

He knows by now whether he is running or not. If he doesn’t know, than I suggest he not run, because if he is that indecisive, then we would be better off with a Governor who is a bit more decisive and can effectively lead us.

I don’t know what is holding Chris Christie back. It could possibly be part of some an ingenious strategy that I am missing?

Personally, I think it is a mistake.

We need to get the ball rolling here in New Jersey. We can’t afford to waist time.

The possibility of Chris Christie entering the race is freezing things in place for Republicans.

Some potentially strong candidates are foregoing their own bid for the nomination in deference to Christie.

People like Morris County Freeholder John Murphy is one of them.

If Murphy is not running for Governor he could become a candidate for the state assembly, a successful one that could help increase our lagging numbers in that chamber. If Chris Christie doesn’t run for Governor, Murphy, who came in third in the last gubernatorial primary, could make a significant run for the nomination this time. Such a move on Murphy’s part would mean that we need to gear up the candidacy of someone else to run for that assembly seat in Murphy’s place.

There are many other pieces that are being prevented from falling into place due to Christie’s indecision.

So I for one suggest that Mr. Christie thaw out the freeze that he has created.

As a former U.S. Attorney, one of the most successful in our history, he knows that long deliberations are often the results of poorly presented cases. As such, if he needs to deliberate over this decision much longer than he already has, than I can only assume that there are too many obstacles in his way.

It makes we wonder what those obstacles could be. Are their unrevealed skeletons in his closet? Do Democrats have something on him?

Whatever the reason Chris Christie may have for prolonging his deliberations, it only creates doubts about him and that is not good.

He needs to give us voters, the jury in this case, his verdict sooner rather later.

If he wants to be our nominee for Governor, he better start creating a sense of confidence in those of us who he wants voting for him.

Right now, his procrastination is showing me that he doesn’t want the job of Governor all that much and the longer he waits to decide, the more convinced I and others become of that. And let’s face it folks……this is going to be a tough election. One that will require our nominee for Governor to want the job, bad enough, to make the sacrifices that will be necessary to get it.

While Chris Christie’s apparent indecision sets in, POLITICS 24/7 wants to know what Republicans are thinking.

So, as of today, who would you like to see be the Republican nominee for Governor of New Jersey?

The following poll lists a few easily possible or likely potential candidates. Take a moment and place your vote.

Results will be released next Monday.

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 TRUE  TALE

When Daniel Arap Moi was the president  of Kenya, Moi wanted a special postage stamp issued with his picture on it.

 

 So, he instructed his people, stressing that it should be of international quality.

 

The stamps were duly released and Moi was pleased.

 

But within a few days of release of the stamp, he began hearing complaints that the stamp was not sticking properly, and he became furious.

 

He called the people responsible and ordered them to investigate the matter.

 

They checked the matter out at several post offices, and then reported the problem to Moi.

 

The report said, “There is nothing wrong with the quality of the stamp. The problem is people are spitting on the wrong side.”


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GRASSROOTS CAMPAIGN TO MAKE JENNIFER BECK LT. GOV. OFF TO A GOOD START

Last week POLITICS 24/7 began planting the seeds of a grassroots effort in the Garden State. It is a campaign to persuade whomever becomes the Republican nominee for Governor to select State Senator Jennifer Beck as their running mate.

For this effort a new blog site was created. It is titled, Jennifer Beck for Lieutenant Governor of New Jersey. Please visit it by clicking on to its highlighted name.

There you will find all the information you need to know why we have started this movement. On the site you will find pages dealing with the campaign, Senator Beck’s voting record, her bio, the latest news in regards to her legislative efforts and this campaign, videos and also the petition which this whole campaign comes down to.

Please be sure to sign the petition urging our eventual nominee for Governor to select State Senator Beck as their running mate and our nominee for Lieutenant Governor.

Also be sure to pass the link on to other New Jersey voters. Simply cut the following url……

http://www.gopetition.com/online/23572.html

…..and paste it into an email to your friends that urges them to lend their support to the cause.

So far the this effort is working. With no money to spread the word, I am relying on word of mouth, the Internet and earned media. To date we have been contacted by The Star Ledger, The Asbury Park Press, The Two Rivers Times, The Philadelphia Inquirer, Politickernj.com and numerous blog and news oriented web sites. Each have, thankfully, published positive stories about the campaign and there will be more coverage to come.

Currently, our efforts are focused on establishing direct contact with voters through the Internet.

In that vein, we have expanded our effort to Facebook.com, where we started a Jennifer Beck For Lt. Gov. group.
You can find it and join it by going to the following link:

http://www.facebook.com/group.php?gid=41874237142

Since yesterday, by taking this route, 42 new, interested and excited members have joined the campaign and many have signed the petition.

We have also created another group platform on an Internet site recently created by Patrick Ruffini, a brilliant Republican, strategist and computer whiz. It is called Rebuild The Party and can be found at rebuildtheparty.com .

On Rebuild The Party is our own group for Jennifer Beck:

http://rebuildtheparty.ning.com/group/statesenatorjenniferbeckforlieutenantgovernorofnew

There too, you can get involved and be a part of this effort to shape a winning, gubernatorial ticket for Republicans in November of ’09.

If you would like to play a role in the petition campaign for Jennifer Beck, your efforts will be greatly appreciated. We could use individuals who are familiar with the Internet. I would really like someone to head up an outreach effort on MySpace.com.

With no financial budget, this grassroots campaign has to sprout up wherever possible and places like MySpace are just right for us. They’re free!

In time, our campaign will be looking to appoint county coordinators. These individuals will help to scoop up all the signatures for our on line petition that they possibly can in their counties. that type of focus will help to maximize our efforts. But that will come further down the long road ahead of us.

In the meantime I truly hope to hear from you and thank you for your interest in the campaign. If you have any questions or would like more information about this campaign for Jennifer Beck, please feel free to reach me at LiberalsRlosers@aol.com . It is an easy email address to remeber. That’s liberals, R, losers, at aol.com.

Hope to see you soon!

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