Top Tweets for #Propionicacidemia
Happy to share our new article!!!!
Metabolomic analysis in hiPSC-cardiomyocytes from propionic acidemia patients
@pa_foundation @guofangzhang @CBM_CSIC_UAM @CIBERER @UAM_Madrid
#propionicacidemia #iPSC #raredisease #metabolomics
https://t.co/uyDjiMcn68
The Future Of mRNA Medicines - Dr. Rose Loughlin, Ph.D. - Executive Vice President, Research, @moderna_tx @ProgressPotent1 #RoseLoughlin #Moderna #mRNA #MessengerRNA #CancerVaccines #NeoantigenTherapy #Melanoma #RareDisease #IntercellularTherapeutics #PropionicAcidemia #MethylmalonicAcidemia #CMV #Cytomegalovirus #EpsteinBarrVirus #ProgressPotentialAndPossibilities #IraPastor #Podcast #STEM #Innovation #Technology #Science #Research https://t.co/ZhrsNCkTmn
Happy December! Our PA girls are featured in our 2024 OAA calendar for the month of December! Our community reaches all over the globe! π#propionicacidemia #allinthistogether
Day 2 of the #simd2024 - We are grateful to pharma partners like @moderna_tx working on new treatments for our rare disorders. #propionicacidemia #MethylmalonicAcidemia
The PA families of the OAA are extremely grateful for Moderna's research in finding a better treatment to manage our children. Thank you so much! #propionicacidemia #allinthistogether #organicacidemia
Today, we announced that interim data for a first-in-human, phase 1/2 study of mRNA-3927, an investigational #mRNA #therapy for propionic acidemia (PA), has been published in Nature. This is the first published clinical data utilizing mRNA therapy for intracellular protein replacement.
mRNA-3927 is a novel investigational mRNA therapy that encodes for the PCC enzyme, and has the potential to replace a missing or dysfunctional enzyme.
The premise has always been that an mRNA-based approach to making #medicine could advance at the pace of information β and we believe that our mRNA platform could change how medicines for rare diseases may be delivered in the future.
Read more: https://t.co/M9bTWlnXa3
https://t.co/IG33d3jzR9
When we learn of another child born with our disorders we give encouragement and support. Brings back memories for our families as we have walked in similar shoes. #allinthistogether #propionicacidemia
Our PA boys are on stage for the 2024 March OAA calendar page! Itβs show time! π¦β€οΈ #propionicacidemia
Happy #RareDiseaseDay My daughter was born with a rare disorder called #PropionicAcidemia 35 years ago. Not much was known then. I'm fighting for those like her and other rare #organicacidemias to come up with better treatments! She's my hero and so very brave!
Exciting news on future mRNA therapies for protein replacement in otherwise untreatable diseases!
#PropionicAcidemia
I wonder if #Antivaxxers will also oppose this if they were the diseased?
We recently reported on interim data from the Phase 1/2 trial of mRNA-3927, an investigational #mRNA therapy for propionic acidemia (PA). The data was presented at the 2023 @ASGCTherapy Annual Meeting. Read more: https://t.co/pWoRWIvEk6
#ASGCT23 #PropionicAcidemia
Exciting news for this potential new treatment for #propionicacidemia
We recently reported on interim data from the Phase 1/2 trial of mRNA-3927, an investigational #mRNA therapy for propionic acidemia (PA). The data was presented at the 2023 @ASGCTherapy Annual Meeting. Read more: https://t.co/pWoRWIvEk6
#ASGCT23 #PropionicAcidemia
We recently reported on interim data from the Phase 1/2 trial of mRNA-3927, an investigational #mRNA therapy for propionic acidemia (PA). The data was presented at the 2023 @ASGCTherapy Annual Meeting. Read more: https://t.co/pWoRWIvEk6
#ASGCT23 #PropionicAcidemia
We are excited to represent our community in San Antonio at the @AbbottNews Metabolic
conference. #organicacidemia #Methylmalonicacidemia #propionicacidemia #isovalericacidemia #glutaricacidemiatype1
Grateful for this research in #propionicacidemia
Today, we announced that an abstract reporting on interim data from the Phase 1/2 trial of mRNA-3927, an investigational #mRNA therapy for propionic acidemia (PA) has been accepted for presentation at the 2023 American Society of Gene + Cell Therapy (ASGCT) Annual Meeting being held May 16-20, 2023 in Los Angeles, CA. Read more: https://t.co/bHm6iMzM0E
#ASGCT23
PaVe GT: A Project from the NIH Aimed at Speeding up Gene Therapy Development https://t.co/uuccwm47Dl #GeneTherapy #PaVeGT #PropionicAcidemia #NIH #NCATS
OAA shares updated information on potential new treatments and clinical trials to our families. Itβs an added bonus when a family comes to my town for the clinical trial! Meet up times to connect is what itβs all about! #methylmalonicacidemia #propionicacidemia #clinicaltrial
Great to hear OAA medical advisor Kim Chapman MD share her experiences with small molecule therapy for MMA/PA #SIMD2023 #propionicacidemia #methylmalonic #hopeforourkids
Today I shared what itβs like being a caregiver to a child with #propionicacidemia to employees at @BridgeBioPharma Thank you for inviting me and creating awareness on this #RareDiseaseDay2023
Hereβs my hero! My reason for pushing for new treatments! New treatments for our warrior patients! #RareDiseaseDay #Propionicacidemia
I was honored to share my journey to the employees at @moderna_tx . We are hopeful to the success of their new treatment for #propionicacidemia
In the third video in our #VoiceOfThePatient series, @KathyStagni, Executive Director of @OrganicAcidemia Association and mom to Melissa, who lives with Propionic Acidemia, shares her passion to connecting families with rare #metabolic disorders.
#RareDisease
Parentβs voices are so powerful- we are grateful to Honey for sharing her family experiences living with #propionicacidemia
In the second video in our #VoiceOfThePatient series, Honey Stecken, mom to Maren, shares her experience of raising a child with Propionic Acidemia (#PA) during #COVID19, and how some parents actually felt safer during the pandemic..(1/2)
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