Antisense trial for SALS on horizon

Nikki J

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Joined
Mar 22, 2012
Messages
8,920
Reason
PALS
Diagnosis
04/2014
Country
US
State
MA
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Boston
As many of you know antisense genetic therapy is hopeful for FALS. The SOD1 trial is in phase 3. C9 is in a phase1-2. FUS is coming to trial in the next year

however Ionis has an antisense targeting ATXN-2-TDP-43 which is found in SALS ( and many FALS). They SAY they expect a trial to start in the latter part of this year. My experience is that it may well take longer but this is incredibly hopeful news for SALS
 

Anjum nazir

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Joined
Dec 31, 2017
Messages
25
Reason
PALS
Diagnosis
07/2018
Country
IN
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JK
City
Srinagar
N what about people like me who are fals but do not know the exact gene involved ...what should we people do even without hope for future therapies
 

Nikki J

Moderator
Joined
Mar 22, 2012
Messages
8,920
Reason
PALS
Diagnosis
04/2014
Country
US
State
MA
City
Boston
You might have the TDp-43 issue. Most PALS do including a lot of FALS. However this is a proposed phase 1 trial that has not yet started not a treatment that is being given.
 
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