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Pat Furlong
  • Female
  • Middletown, OH
  • United States
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Pat Furlong's Friends

  • Greg
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Pat Furlong's Discussions

In Memory of Shelley Burks White

Started this discussion. Last reply by Colleen Jun 11, 2009. 6 Replies

15 things about your family that make you smile

Started this discussion. Last reply by Christine Piacentino Apr 25, 2009. 8 Replies

 

Pat Furlong, Founding President & CEO

Profile Information

About me:
President/CEO of PPMD. In my earlier life, MS in Nursing, experience primarily in ICU, CCU, Dialysis, ER . Later taught nursing education and patient education.
Interests - DMD, insuring all of the pieces of treatment and cure are in place.

Read my Bio: http://www.parentprojectmd.org/site/PageServer?pagename=About_media...
About my family:
4 children. Chris and Patrick were diagnosed in 1984, the 'no hope and no help' era that thankfully is OVER.
Jen and Michelle, daughters, now live in Philadelphia and New York.
Husband is a physician.
Name(s) of child(ren)/individual(s) with Duchenne:
Christopher and Patrick
City:
Middletown

Pat Furlong's Blog

PTC Therapeutics Provides Regulatory Update on Translarna™

Posted on July 25, 2016 at 12:00pm 0 Comments

PTC recently participated in discussions with FDA to discuss the Refuse to File (RTF) letter issued on February 22, 2016 with respect to the company's New Drug Application (NDA) for Translarna for the treatment of Duchenne. Today PTC…

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BioMarin Discontinues Clinical Development of Drisapersen

Posted on June 1, 2016 at 9:57am 2 Comments



Today is June 1 and I know many of us feel like 2016 has beat up this community already. With every small victory, we face another major setback.

 

As you have heard by now,…

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Every single carrier.

Posted on May 9, 2016 at 10:07am 4 Comments

I am a genetic carrier of Duchenne. My mother was not. This means I was one of the 1:4600 females born with this genetic mutation. As a child, I had muscle cramps, ‘growing pains’ my parents would say. And from those ‘growing pains,’ 20 years later, my sons were diagnosed and I first learned…

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Changing the Paradigm: The Eteplirsen Ad Comm

Posted on April 25, 2016 at 10:30pm 5 Comments

There is no way around it, today was hard. Hundreds of us – the largest gathering of Duchenne families in history – came together to tell the FDA that eteplirsen is safe, effective, and should be approved using the tools…

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Growing Our Team

Posted on February 1, 2016 at 9:43am 0 Comments

We are not a team because we work together. We are a team because we respect, trust, and care for each other. – Vala Afshar

 

I am the first person to admit that I don’t know much about a lot of different things. But I do know talent when I see it. And I could not be…

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Latest Activity

Shelly favorited Pat Furlong's blog post PTC Therapeutics Provides Regulatory Update on Translarna™
yesterday

Staff
Pat Furlong posted a blog post

PTC Therapeutics Provides Regulatory Update on Translarna™

PTC recently participated in discussions with FDA to discuss the Refuse to File (RTF) letter issued on February 22, 2016 with respect to the company's New Drug Application (NDA) for Translarna for the treatment of Duchenne. Today PTC announced that they have recently submitted…See More
yesterday
ramon ciriaco viaña ortiz commented on Pat Furlong's blog post BioMarin Discontinues Clinical Development of Drisapersen
"Hola  como  estan  grasias  por  dejar entra a su comunidad un  comentario serca de mii hijo con  duchenne, a  el  lo estaba atendiendo una neurologa  aca en  Cartagena  Colombia de nombre…"
Jul 5
Brid Murray commented on Pat Furlong's blog post Every single carrier.
"Thank you.  Yes, being a carrier is always somewhere is the back of your mind and in your 'backpack'.  I think you are correct Pat.  We are all manifesting in some way or another. "
Jun 9

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Comment Wall (87 comments)

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At 4:57pm on June 22, 2015, Kuldeep Singh said…

Hi Pat

Thanks for all your efforts for PPMD and DMD research
My name is Kuldeep Singh.
My boy is  4 years 3 months old .His name is Neil Singh.
He is our only child.
We are based in Auckland, NZ

He has been diagnosed with DMD last year with exon 41 duplication
We can see that he is going downhill day by day

I saw a webinar for duplication research update
Hopefully it will help Neil in future

Just need your advise on some tricky situation that we have got at moment

We saw a trial starting soon
http://www.catabasis.com/clinical-trials-cat-1004-movedmd.php

Requirements for participating in this clinical trial include:

    Boys with a diagnosis of DMD (any confirmed mutation) between the ages 4 and 7 years
    Ability to walk independently
    No corticosteroid use within the past 6 months and no plans to start corticosteroids in the next 6 months

Our boy will be starting steroids soon

My wife enquired Joanne Donovan, MD, PhD (Chief Medical Officer at Catabasis) about Neil's participation in trial

We got a response by email

"Regarding your son and potential participation in the MoveDMD trial, at
this time, our regulatory approval and conditions limit us to the U.S.
only.  This is confounded by the fact that patients residing outside the
U.S. may not be permitted to travel back and forth with investigational
product. While some circumstances exist, we are currently working through
how, if at all, we will be able to support and enrol potential patients
who reside outside the U.S.  I know this is not the news you want to hear
but would like to keep in touch with you should circumstances change."

Joanne was kind enough to call us and discuss it,her concern was if we will be
able to take any medication to NZ after trial

When we asked our Neurologist has advised to suggest
"That your doctor is supportive and that I could negotiate with NZ regulatory
bodies to try to ensure the medications could enter New Zealand and feel confident I could make this happen."

We are really confused about this situation
Neil is 4 years and 3 months now

We want him to get any possible treatment if it can help to save him

On the other end we are not sure if we should start his steroids or not as
we are at the verge  of starting them

It is really different here in NZ as compared to US in terms of awareness about Duchenne, trials etc
There is no dedicated Duchenne Centre here

Doctors are very helpful however  most of times  we are scared about our boy
Whenever we see our Neurologist, we get more confused

Can you please advise your thoughts about this as I believe that you
may have an answer for this

At 1:50pm on June 18, 2012, Tammy said…

Hi Pat,

Do you know if there have been any studies on DMD boys and enzyme deficiencies?  I met with a 5th generation doctor from India that says this is very common, and a cause of many problems with muscular dystrophy patients.

Thanks in advance...

Tammy

At 6:05pm on April 11, 2012, stephen beggs said…

hi pat, just want to let u know that i am finding ppmd an absolutely brilliant resource and support. since my lovely wee nephew ben was diagnosed recently with duchenne, i feel as if i have become an expert on neuromuscular conditions. But we have to, as we have to take control for our boys. I think that you are an inspiration for all that you have achieved so far with pushing duchenne research abd treatment to where we are today. it is looking so much more promising. I recently sent u a friend request and would so appreciate to have u as a friend. God bless you pat and all your team. stephen beggs.

At 2:55pm on December 10, 2011, Lisa Guest said…

Hi Pat, I am happy for you to use photo of my boys. I also got an email fron Wil Nolan for the same request. Wishing you and your family a Merry Christmas...

Lisa Guest

At 10:14am on December 6, 2011, adnan sarwar said…

I m Adnan Sarwar from Pakistan. i m suffering from muscular dystrophy 

i m intrested to get some new research information and meet with other

md and their family for discuss issues related md.pls help me in this

connection.

At 2:46pm on May 15, 2011, Julie Leston said…

Dear Pat,

Thank you so much for responding back. I would love to speak with you. What time tomorrow (Monday) would be a good time to talk to you? My phone # is 847-856-1201 and email address is momsangel0507@aol.com. I do have your cell phone # and will be happy to call you anytime you are available. Take care and thanks again.

 

 Sincerely,

 Julie Leston

At 11:27pm on March 16, 2011, Audrey Tan said…
Pat was wondering have u heard about Dr.Rhodes in corpus christi texas reagarding the sts vecttor treatment?
At 2:03pm on January 24, 2011, Suzanne Desmond said…

Hi Pat,

I just wanted to let you know how happy I am to hear about the Super Bowl & the awareness that it will bring.  I also wanted to let you know that my Minister Rev. Richard Young spoke of you & the article of you & PPMD in the New York Times.  He said so many nice things about you & how your story touched his heart.  His sermon was called "When God comes down".  After the service I gave him a hug & thanked him for speaking about DMD & let him know that he has brought awareness to our congragation.  Just wanted you to know that.

God Bless,

Suzanne Desmond

Orchard Park, NY (Buffalo, NY)

At 7:58am on January 8, 2011, Gerardine Gardezi said…
Thank you Pat ,for replying.As it is all new to me i am slowly trying to assimilate information and this site is a great help.I read in a few places many mums talking about a carrier dvd that is available.Where can i access this?My family as a whole need advice and education as to how best to support my sister and her family.Enable Ireland is my next port of call this month.They will play a major role in the care of my nephew.All the talk of figures etc i find confusing.....different depletions,etc.I hope i will learn as i go along and the support i witness here is brilliant.My eyes have been opened so much here and i don't feel as lost as i was.At the end of the day it is parents and their children here that will guide us best i feel.Thank you for your time,Gerardine.
At 9:07pm on December 18, 2010, Char Burke said…

Hi Pat - Just read the article in The New Yorker.  Excellent article.  And whoever wrote it, sure did alot of background work.  Really enjoyed it.

Happy birthday!  I hope this year is the best year yet for you!  I hope Tom is doing well too and of course, your girls.  Thank you for your leadership, your tenacity and for all you do - Best -Char Burke

 
 
 

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