Rafael

Medical Affairs Leader for Interested in #RareDiseases #Neurology #Liver #Hepatology #Bone

[Rafael @_RafaelSierra]

If you are at #ESGCT23 join us today at our Symposium “Breaking down barriers bringing the benefits of gene therapy to patients” with Dr Luca D’Angelo and Prof Wuh-Liang (Paul) Hwu at 13:25-14:25 in Shed 2b #genetherapy #AADCdeficiency

[Rafael @_RafaelSierra]

Definitely agree that natural history studies help advance #raredisease treatments. They help patients, families, clinicians, regulators, payers, policy makers understand the disease and make informed decisions.

[PTC Therapeutics @PTCBio]

Learn more about natural history studies and why they are so important in the study of #rarediseases and development of potential therapies: https://t.co/MCkdeNGkM6

[Rafael @_RafaelSierra]

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

[Rafael @_RafaelSierra]

Very interesting session by Dr Sheila Wong from HK Children’s Hospital at #AOCCN2023 sharing how important it is to not miss #neurometabolic and #inheritedmetabolic disorders, including #AADCd.

[PTC Therapeutics @PTCBio]

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

[Rafael @_RafaelSierra]

Excelente oportunidad para escuchar como lidear cuando alguien en la familia es diagnosticado con una #enfermedadrara.

[PTC Therapeutics @PTCBio]

Does your brother or sister have a #raredisease? Join us at 7 p.m. EDT tomorrow, July 26, as we spotlight your important role. This webinar will be held entirely in Spanish. To register, please visit: https://t.co/sG8PlkVsUk

[Rafael @_RafaelSierra]

@"AADCAware Registry: an example of the value that disease registries bring to #raredisease #AADCdeficiency"https://t.co/sWn4dC6HOk on @LinkedIn

[Rafael @_RafaelSierra]

Look forward to moderating @PTCBio’s #AADCdeficiency symposia at #AOCCN2023 in Bangkok. Unique opportunity to hear from Prof Hwu, Dr Chien and the Poulin Family of their #raredisease #AADCd experience.

[PTC Therapeutics @PTCBio]

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

[Rafael @_RafaelSierra]

Amazing to see how PTC’s innovative science is contributing to help patients living with #raredisease including #ALS.

[PTC Therapeutics @PTCBio]

We’re proud to be featured in a recent article from @NatureBiotech. “Unprecedented blood biomarker enables ALS drug approval” includes PTC’s approach to #ALS, highlights the ongoing CARDINALS trial and shares insights from our CEO Dr. Matthew Klein.

[Rafael @_RafaelSierra]

It was great too see so many colleagues and friends today at the #EPNS2023 PTC #AADCdeficiency symposia. Great presentations from Prof F Ezgu, Prof T Opladen, and Dr H-M Lee. Focused on multidisciplinary teams working together to help kids with #raredisease #AADCdeficiency

[Rafael @_RafaelSierra]

Counting the days to #EPNS2023 to see my colleagues, investigators, and friends. I am proud of the scientific posters and activities our GMA team has put together to advance #raredisease #AADCdeficiency awareness.

[PTC Therapeutics @PTCBio]

We’re looking forward to #EPNS2023 to help advance research and knowledge of rare neurological conditions like #Duchenne muscular dystrophy, #AADCdeficiency and mitochondrial disease. PTC has a number of activities planned, including two symposia, two booths and numerous posters.

[PTC Therapeutics @PTCBio]

We’re looking forward to #EPNS2023 to help advance research and knowledge of rare neurological conditions like #Duchenne muscular dystrophy, #AADCdeficiency and mitochondrial disease. PTC has a number of activities planned, including two symposia, two booths and numerous posters.

[Rafael @_RafaelSierra]

https://t.co/FZQNyr3u49

[Rafael @_RafaelSierra]

Looking forward to an exciting evening at #PrixGalien Awards Ceremony with the UK @PTCBio team.

[Rafael @_RafaelSierra]

Cannot wait to join my ⁦@PTCBio⁩ UK colleagues at the #PrixGalien Award Ceremony on Thursday.

[Rafael @_RafaelSierra]

I’m so proud to be part of the Global #GeneTherapy team at @PTCBio that works everyday to bring innovative treatments to patients with #rarediseases

[PTC Therapeutics @PTCBio]

We're honored to be selected as a finalist in the 2023 Prix Galien UK Award for "Best Pharmaceutical Product." We look forward to joining colleagues and other finalists at the award ceremony on May 11 in London. Read more: https://t.co/Afb5f8QEtR

[PTC Therapeutics @PTCBio]

Live from Brussels! #EconCellGeneTherapySummit #genetherapy #raredisease

[PTC Therapeutics @PTCBio]

We're honored to be selected as a finalist in the 2023 Prix Galien UK Award for "Best Pharmaceutical Product." We look forward to joining colleagues and other finalists at the award ceremony on May 11 in London. Read more: https://t.co/Afb5f8QEtR