BeritSofie
  • Female
  • Orkdal
  • Norway
Share on Facebook
Share

BeritSofie's Friends

  • Blazena Kaderabkova
  • Bernardo A. Iriberri
  • neo
  • Pavlina
  • Teresa Buchanan
  • Angela
  • Char Burke
  • David Feder
  • Julie Garcia
  • PPMD
 

BeritSofie's Page

Latest Activity

BeritSofie replied to Steven's discussion PTC gets EU approval
"My concern now is that the EMA will follow the CHMP's decision, and not give approval for the boys not walking. Then we have to wait for  3 more years. Our boys will be weaker, and perhaps for some - too late. We have to have the parents…"
Jun 13, 2014
BeritSofie replied to Steven's discussion PTC gets EU approval
"Hello all I think this is good news too, BUT once again the non ambolatory boys are forgotten ! The CHMP's members will recommend that EMA (european medicines agency) will give approval to boys from the age of 5 and AMBULATORY.  Every boy…"
Jun 10, 2014
BeritSofie replied to Bernardo A. Iriberri's discussion Ataluren Webinar
"Hi Pat and Sharon Very important recommendations:  http://www.parentprojectmd.org/site/DocServer/WhitePaper_2013FINAL.pdf?docID=13883&AddInterest=1821 Will this help speed up approval for Ataluren ? Will the European Medical Agency also…"
Apr 23, 2013
BeritSofie replied to Bernardo A. Iriberri's discussion Ataluren Webinar
"Thank you Sharon. Please keep us posted  - good or bad news. Would also like to hear what PTC says, when you have spoken to them about the time line. Needless to say, all boys with a stop codon not included in one of the previous Ataluren…"
Mar 18, 2013
BeritSofie replied to Bernardo A. Iriberri's discussion Ataluren Webinar
"Thanks Sharon Although we live in different contries around the world, I don't think there are differences in how parents relate to the risk/benefit issue. We all have one thing in common, not matter where in the world you live. We want the…"
Mar 16, 2013
BeritSofie replied to Bernardo A. Iriberri's discussion Ataluren Webinar
"Hi Sharon Thank you for your reply. This paper you are working on, will it adress Ataluren specific or trials in genereal ? You mentioned earlier that having the parents voice up front when it comes to what we are willing to risk contra benefit…"
Mar 14, 2013
Bernardo A. Iriberri left a comment for BeritSofie
"Hello Berit. Thank you very much for your invitation. I hope we can do something to wake up those involved in the approval process, but they all seem to be entangled in a very bureaucratic approach, and they fail to see what is obvious: that the…"
Mar 2, 2013
BeritSofie replied to Bernardo A. Iriberri's discussion Ataluren Webinar
"Hi Bernardo, and one more question to Sharon Share you fear for the timeline, Bernardo. Not sure how to lobby the EMA, but Sharon mentioned earlier that having the parents voice up front when it comes to what we are willing to risk contra benefit…"
Feb 28, 2013

Profile Information

About me:
Mother of a 12 year old boy with DMD
Name(s) of child(ren)/individual(s) with Duchenne:
Magnus
Age(s) of child(ren)/individual(s) with Duchenne:
7-12
City:
Orkdal

Comment Wall (9 comments)

You need to be a member of PPMD Community to add comments!

Join PPMD Community

At 6:43pm on March 2, 2013, Bernardo A. Iriberri said…

Hello Berit. Thank you very much for your invitation. I hope we can do something to wake up those involved in the approval process, but they all seem to be entangled in a very bureaucratic approach, and they fail to see what is obvious: that the Phase IIb trial was a success and that it shouldn't have been terminted. I am sorry about your personal case, with all the preparations for the trial and the sudden cancellation, etc. Well, let's keep insisting, and see if we can do something. Best wishes. Bernardo 

At 7:19am on April 7, 2011, Blazena Kaderabkova said…
Now is Genzyme under sanofi-aventis and may it is not clear if sanofi will continue in ataluren development. It is very frustrating.
At 11:38am on January 25, 2009, Sharyn Thompson said…
We are actually Australian, but just recently moved to London (after 8 years in SIngapore and Tokyo). My husband has organised a bike ride from the Neuromuscular INstitute in Sydney to the Neuromuscular institute in Melbourne and will in fact have the head professor from the melbourne institute and another guy from the Sydney institute accompanying him. I am also in contact with the Sydney doctors coordinating the trial, so I might send off an email to them tongiht and ask. I'll let you know what they say. wouldn't it be great if they were still recruiting????
At 6:33am on January 25, 2009, Sharyn Thompson said…
Quick question, are they still recruiting in Australia? I know it's a long way but it could be an option. I know the UK trial is full and so we are going to see if Australia is full and try to get James on it when he turns 5 in May. We'll do anything for him, just like you would. It was just a thought....
At 9:23am on January 7, 2009, Sharyn Thompson said…
Hi Berit,

I have only today read your discussion on trying to get your son into the PTC124 trial. I was wondering if you ended up getting him on it? Our son is 4.5 and has a nonsense mutation, but of course he was too young for the trial. I am so sorry Magnus didn't get on the trial and I am hoping you got him on somewhere, as i somewhat understand the sadness and frustration of not being included.

All the best,
Sharyn.
At 2:44am on December 31, 2008, JUAN PEDRO ARBULU said…
the best luck for you on your way to the trial!!
At 1:49pm on November 29, 2008, Angela said…
Hi Berit,

The list I posted has other sites in Europe also. If I were you I would start there. Jack has been part of the 2a extention for 3 months and the travel has been much harder on him than expected. We only fly 1 1/2 hours to Utah for treatment. Though I believe the 2b is less demanding, it is still a serious consideration. Best of luck to you and Magnus, I hope you are able to get him in the trial.

He is a real cutie!!

Ang :)
At 12:13pm on November 29, 2008, Julie Garcia said…
Berit,

Magnus is very handsome!

Julie
At 12:03pm on November 29, 2008, Julie Garcia said…
Hello Berit!

I hope all is well and look forward to seeing pictures.

Take care,

Julie
 
 
 

Need help using this community site? Visit Ning's Help Page.

Members

Events

© 2019   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service