Top Tweets for #AADCDeficiency
🧬 New publication from GTI researchers!
A new study explores an expanded #genetherapy delivery approach for #AADCDeficiency by targeting both dopamine- and serotonin-related pathways in the brain. 🧠
https://t.co/jY38jFGdRN
We're honored that our #genetherapy treatment for #AADCdeficiency received the Therapeutics Innovation of the Year by BioTech Breakthrough Awards, a milestone in advancing care for patients with a rare disease.
เร่งระดมทุนช่วยเหลือน้องอคิน : ค่ารักษา 107 ล้าน สำหรับเด็กน้อยผู้ป่วยโรคหายากหนึ่งในล้าน AADC Deficiency ผ่านการรักษาด้วยยีนบำบัด https://t.co/pi6Qr9QEIU
#โรคหายาก
#AADCDeficiency
#สภากาชาดไทย
The FDA's approval of #Kebilidi is a breakthrough for #AADCdeficiency patients, bringing new hope! 🌟💉 🌍✨ More at https://t.co/UFa27rXs9p
#GeneTherapy #MedicalInnovation #Neuroscience 🧠 #HealthcareForAll ❤️
Today, Judy Wei and I celebrate our daughter’s 'reborn day'—exactly five years since she received the life-saving #GeneTherapy for #AADCDeficiency. Since then, her incredible progress has transformed our lives from full-time care to a journey filled with learning, traveling, and exploration.
Watching her thrive has been our greatest gift, inspiring us to support others on a similar path. Through free educational resources, published books, research articles, and advocacy, we’ve worked tirelessly to raise #awareness and push for global access to treatment. We’re thrilled that our advocacy played a small role in the approvals in the EU, UK, Taiwan, and now, incredibly, the FDA—on the very day of her reborn celebration.
This #FDAApproval marks a hopeful new chapter for future families with access to this groundbreaking treatment and a supportive community to guide them. Our deepest thanks to PTC Therapeutics, Inc., the doctors, and the researchers whose dedication turned this ‘science fiction’ treatment into a beautiful reality.
Today, we are grateful and honored to share this journey with all of you. 💙
#BreakingNews: Today, we are proud to announce that the FDA has approved our #genetherapy for treating adults and children with #AADCdeficiency. Learn more: https://t.co/6QShxigmdt
#BreakingNews: Today, we are proud to announce that the FDA has approved our #genetherapy for treating adults and children with #AADCdeficiency. Learn more: https://t.co/6QShxigmdt
We are proud to mark AADC Deficiency Awareness Day today, together with our partner PTC Therapeutics.
It has been an honor to help raise awareness of this rare pediatric genetic disorder. #CLPT #AADCdAwarenessDay #AADCdeficiency #Nsgy
https://t.co/4ejHvhV15q
We’re proud to be attending #SSIEM 2024 in Porto, Portugal. We invite healthcare professionals attending the conference to join us for our sponsored symposium sessions focused on #AADCdeficiency and #PKU. #ExploringNewHorizons #Porto
We’re proud to be a part of the collective efforts to advance research and knowledge about #AADCdeficiency. Congratulations to all on this important publication.
We are proud to help publish this paper with passionate healthcare professionals. #Raredisease patients are benefiting from #genetherapy. We will support others in their journey to care for their children, access resources, support research, and advocate for awareness: https://t.co/4arBEzExy5
Stop by booths 529 and 531 to meet our team at the @AmEpilepsySoc Annual Meeting! Learn about #genetictesting for patients with suspected neurotransmitter disorders, as well as #AADCdeficiency as a potential underlying diagnosis in patients with suspected seizures. #AADCd
HCPs are invited to attend Session 12 in Shed 2A on Friday entitled "From Advanced Translational Studies to Approved Therapies," where Prof. Hwu will present his findings from long-term clinical trials for #AADCdeficiency at the @ESGCT 30th Annual Congress.
Please join our partner @PTCBio to learn how #CLPT tech is used to treat young children w/ #AADCDeficiency in the EU & clinical trials in the US at today’s Lunchtime Symposium - Breaking down barriers: Bringing benefits of gene therapy to patients - 13:25 in Shed 2B #ESGCT2023
#AADCDeficiency is a rare disease that leaves children very dependent and fighting for life. Fortunately, Rylae-Ann had life-saving gene therapy for #AADCd. She has made amazing progress by working hard. Today, she has a message for AADC families. https://t.co/LyWUCRNOtt
We joined #Taiwan Foundation for Rare Disorders & National Taiwan University Hospital for an educational event at a facility dedicated to #raredisease. The event hosted therapists, doctors, & researchers to provide support for #AADCdeficiency families https://t.co/2bJcL4Ftib
A fellow #AADCdeficiency family shares how innovation and collaboration between @PTCBio and @ClearPointNeuro brought about life-changing results for their child: https://t.co/TTmfiXNok8
Many thanks to Prof Paul Hwu & Dr Nancy Chien (@NTU_Hospital) and Richard & Judy Poulin ( @edu4rare) for presenting at PTC’s #AADCdeficiency symposia. #AOCCN2023 #raredisease
Here are a few reasons @_RafaelSierra is looking forward to moderating our sponsored symposium on #AADCdeficiency with Prof. Hwu, Dr. Chien and the Poulin family. Join us on August 5 in Bangkok, Thailand. #AOCCN2023 #AADCd
Gain insights on diagnosis and the impact of #AADCdeficiency from experts and caregivers at our symposium on August 5 at the 16th Asian Oceanian Congress of Child Neurology (AOCCN). #AADCd #AOCCN2023
Delighted to share how ClearPoint's MRI-guided platform empowered direct delivery of gene therapy to the brain of a 3-year-old little boy. Simone underwent the 1st commercial #AADCDeficiency #genetherapy procedure in Italy performed @PoliclinicoUmb1 #CLPT #Nsgy #SmartFlow
Hear the remarkable story of 3-year-old #AADCdeficiency patient Simone. In May, he underwent the 1st commercial brain gene therapy procedure in Italy using ClearPoint’s #SmartFlow cannula w/ our MRI-guided navigation platform. #CLPT #GeneTherapy #Nsgy - https://t.co/XtvxLsSJdF
Hear the remarkable story of 3-year-old #AADCdeficiency patient Simone. In May, he underwent the 1st commercial brain gene therapy procedure in Italy using ClearPoint’s #SmartFlow cannula w/ our MRI-guided navigation platform. #CLPT #GeneTherapy #Nsgy - https://t.co/XtvxLsSJdF
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