Top Tweets for #mps3a
$SRPT Conner the 1st kid treated with microD GTx SRP-9001
Jamison with #LGMD2E
Lulu with #MPS3A
Billy our champ living with DMD & still walking thanks to #Exondys51
$SRPT LYS-SAF302 GTx in #MPS3A Ph2/3 study enrolled 15 patients
Completing dosing in 20 patients in H1-2020
Great to see that $SRPT & partner Lysogene already dosed 9 #MPS3A patients out of 20. Very good progress in this tough rare disease, where #GeneTherapy might be the best treatment to those kids!
$SRPT & Lysogene Ph2/3 AAVance study in #MPS3A fully on track with 7 children already safely treated with LYS-SAF302
https://t.co/BDc5tbm0Tc
$SRPT's partner Lysogene treated 3rd patient in the #MPS3A trial
Lysogene Announces first European patient treated in AAVance, Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A)
https://t.co/f20SzORK2r
You won't believe it, if you don't watch this video of Lulu riding bike post her #MPS3A Gene-Therapy by Lysogene, $SRPT's partner, 9wks still hardly moving & need help & to racing after 15 weeks 👏
https://t.co/oeOD1zavEz
Overwhelmed, humbled and grateful to work alongside these generous people.
Norwood Family, thank you for loving me and supporting my precious niece @hopeforshug today on National #MPSawarenessday 💜 #curesff #MPS3A @CureSanfilippoF
$SRPT & Lysogene GT clinical trial in #Sanfilippo #MPS3A
Lulu's mom share her progress post #GeneTherapy treatment
11 weeks: Lulu’s school re-evaluation, in the past year Lulu has shown progress in every single category. Most importantly her cognitive age increased by 6 months
$SRPT & Lysogene Gene-Therapy clinical trial in #Sanfilippo #MPS3A
Lulu's mom share her progress post #GeneTherapy treatment
9 weeks after GT: Lulu's vocab had a second wave of explosive growth. She learned 9 words this week:
And she can ride a bike 👏
Wow! $SRPT doing the Lord's work. God bless this little girl and other #MPS3A patients. This is why so many investors have no ambition to sell. They only trust DI and his team to get it done! ---thlizrd🦎
https://t.co/xPwIGQdGRR
$SRPT & Lysogene Gene-Therapy clinical trial in #Sanfilippo #MPS3A
The mother share here daughter Lulu progress post GT treatment
5 weeks after GT: Faster. Flat footed. Knee & foot centered, More upright/not crounched
https://t.co/eAGKiktO0p
Lulu is the 1st #MPS3A patient treated by Lysogene & $SRPT
Lysogene and Sarepta Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A)
February 14, 2019
$SRPT & Lysogene Gene-Therapy clinical trial in #Sanfilippo #MPS3A
The mother share here daughter Lulu progress post GT treatment
5 weeks after GT: Faster. Flat footed. Knee & foot centered, More upright/not crounched
https://t.co/eAGKiktO0p
@MauriceOnTW @greyzone513 @obryanjf Agree, Don't forget that LysoGene CEO is a mother of #MPS3A girl & she knows first hand what's the QoL means for the kid & the family... '
So, she understand the disease very well & partnering with $SRPT gave them the financial & scientific support as well
Anyone can translate French to English?
Please take a look at this short video of $SRPT's partner for Lysogene working on #MPS3A
For those who speak French. https://t.co/DryLKrkO7g
https://t.co/br7z4VElrv
$SRPT @KarenAiach @LYSOGENE
@Hall8Jack The article writes of her daughter, who benefited from first treatment calling her operation "spectacular". She was less agitated than before, more smiling, she also found sleep, appetite and a calm attitude. Initial trial was around 2011-12.
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