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Firefly

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Thank you Laurie,
I'm keeping my fingers crossed.
 

lovemybrother

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Hi my brother has ALS and he is interested in this as well. Could you provide me information? Thank you Julie
 

diagnosed2016

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What are the odds this will get approved in June? And does anyone know what the timeline to get to market is once approved?
 

lgelb

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The contract sales force has been hired so as to launch soon after approval, presuming approval occurs, which in the current climate I think it will though the data are not a slam dunk.

Note only about 20% of ALS pts are estimated to be in the group this was trialed in (early disease--eat/move/breathe independently, <2y out from dx). So the FDA and therefore PBMs (pharmacy managers for health plans) don't have to approve it for anyone else, but given the logistical barriers to starting an infused drug, and the long drought in ALS therapies, they will likely hold the door open for a while to see what the spend will be, then restrict more as needed.

The PBM pipeline updates are not suggesting that they will limit it to anything past an ALS dx so far. Then again, the FDA is the entity that approves the product labeling, so the PBMs haven't seen it yet, and that can influence what they do.
 

AKNate

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Speaking of the FDA, ALSWorldWide is urging people to send them an email saying "I join ALS Worldwide and the community of ALS advocates in urging FDA to approve the drugs Masitinib and Edaravone as quickly as possible for the immediate benefit of those suffering from this devastating disease."

I don't live in America but will be sending my email now. I'm getting so enraged lately at how slow Australia is, it is so hard to find a doctor who will help us administer the medicine in our city.
 

affected

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AKNate did Dominic Rowe refuse to have anything to do with helping administer? Did he give a reason?
 

AKNate

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I haven't actually spoken to him. He costs $500 to see & in worried about paying that for a rejection again. I spoke to MND NSW & they were trying to tap into him for me to save the cost... I haven't heard anything back. I might try passing on a message through his receptionist. I fired off emails to all the MND associations in Australia last night. I hope someone can help...
 

affected

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I was under the impression you would see him at the MND clinic rather than as a private patient? Good luck, but I can understand reticence to administer IV a drug that is not approved in the country, such a litigious world we are in.

Have you been personally approaching doctors and asking them to administer? How many doctors have you tried? I think approaching the MND associations in all states may be a good way to find some contacts and help. Please keep us posted.
 

AKNate

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The only free clinic I know of is the one we already go to. It's not MND specific but it's neurological. It's at a university. All other neurologists are private.

I've approached 3 GP's and one is trying to find a neurologist through his contacts. All the GP's said no to administering it, they don't have the means in their small clinics plus they don't want to be liable. I don't understand why they make it so hard... our doctor is part of the research board who allowed the first SMA child in Australia to be treated with Biogen/Ionis drug only this month... I don't understand why they can't help us too.
 

MaggieMay

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Just emailed you! :) If anyone wants info - please message me. My hubby is there now getting treatment.
AKNate
Hi I'm new to the forum, my husband has just been diagnosed. We live in Adelaide and are very interested in going to Japan for treatment. Any information would be greatly appreciated re:
Cost
Treatment regimen
Getting a prescription
Where to go?
General experience with medication
Thanks
Margaret
 

AKNate

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Hey Maggie, I just posted a message on your profile. Send me your email address & I'll shoot through what I know.

In terms of script, I haven't found anyone yet but now we can both tackle this problem together!
 

AKNate

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I'm in Japan now with my husband. His grip and pinch test results have been consistent if not improving since he got here - which is amazing. His primary problem is in his legs though however his hands started to play up before he left home in feb.

His pointer fingers & thumb are now weak. Not sure how the grip & pinch tests are stable/improving but those fingers are clearly weak - I can feel the differnece in resistance strength.

Personally I have noticed a little less twitching all over his body but have seen moments when his finger has gone crazy.

His legs seem to be doing ok though, which is a positive!

The doctor gave me some prelim papers that have been drafted before they get released. It says patients on the medicine on average dropped 5 points on the ALSFRS vs 7 on placebo over 24 weeks. These patients were monitored 12 weeks before hand with no medication to create a baseline. I haven't fully read the document yet but when I do, I'll reply with what's interesting.
 

Luesco24

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Apr 18, 2017
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PALS
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09/2016
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FDA already approved it comes with a price, about $145,000 a year. How many of us can afford this treatment.
 

solankinirav01

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May 26, 2017
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05/2016
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Gujarat
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Ahmedabad
Just emailed you! :) If anyone wants info - please message me. My hubby is there now getting treatment.

My uncle was diagnosed with MND last year in May. His progression was fast in initial days then it slowed. But from past 1 month progression has started rapidly again. He is currently on Riluzole but with no significant effect. Can you please guide us about Edaravone. We are living in India, so also if you have any info on how to get it in India it will be very helpful.
Thank you.
 

AKNate

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Messages
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Hi Solankinirav,

I'm sorry to hear about your uncle.

With treatment in Japan, you need to go through a medical guarantor - here is a full list of them: List of Registered Guarantors (Medical Coordinators etc.) (English) <span lang="ja">????????????????????????????????</span> ? Ministry of Foreign Affairs of Japan They will act as your middle man for a once off fee. We used ?????????? | Kokusho International Medical Service

They set up all the appointments for us for 3 months worth (60 drips) at Yoshino Clinic in Tokyo (‹g–ì“à‰È�E�_Œo“à‰Èˆã‰@ ALS ‹ØˆÞ�k�«‘¤�õ�d‰»�Ç ƒŠƒnƒrƒŠƒe�[ƒVƒ‡ƒ“ Žs�ì �‘•{‘ä �¼ŒË). The doctor that my husband sees there is Dr Yoshino himself who was the main Doctor involved with discovering Edaravone/Radicut worked on MND patients. He is such a lovely man and so sympathetic.

My husband does a daily drip of 60mg at the clinic and has a rest on Sunday. The drip takes him about 40mins to go through.

Besides using the Social Med Work or flying to Japan and buying the medicine - I wouldn't know how you would get it in India, i'm sorry.

I hope that helps!
 
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