PTC Therapeutics @PTCBio
PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for patients with rare disease ptcbio.com United States Joined May 2009-
Tweets1K
-
Followers2K
-
Following298
-
Likes1K
Today our leaders participated in the Cantor Global Healthcare Conference 2025 to share the latest updates on PTC Therapeutics. Listen to the webcast to learn more: bit.ly/4mQWvdE
PTC Therapeutics is sponsoring a scientific symposium at #ICIEM2025 in Kyoto, Japan. We invite all HCPs at the conference to join us for a discussion on advancing research and care for children and adults living with #phenylketonuria (PKU).
PTC will participate at the Cantor Global Healthcare Conference, the Wells Fargo 2025 Healthcare Conference, and the Morgan Stanley 23rd Annual Global Healthcare Conference this September. Learn more: ir.ptcbio.com/news-releases/… 
#BreakingNews for #investors and #media: We are proud to announce the U.S. FDA has approved our treatment for children and adults living with phenylketonuria (#PKU). Read the press release for more: bit.ly/4megZvQ
On Thursday, August 7, our leaders will report our second quarter 2025 financial results and provide an update on the company's business and outlook. Learn more: bit.ly/4f8uHy7
Today we proudly recognize International PKU Day, led by @official_espku to spread awareness and education about living with #PKU. Learn how the dietary challenges of this rare condition can impact the quality of life for both children and adults: bit.ly/4kYLPsl
We are proud to announce the European Commission has granted marketing authorization for our medicine to treat children and adults living with phenylketonuria (PKU). Learn more: bit.ly/4k0obdG
Our CEO, Dr. Matthew Klein and CFO, Pierre Gravier will participate in the Goldman Sachs 46th Annual Global Healthcare Conference 2025 today June 10 at 9:20 a.m. ET. Tune in to our fireside chat to hear our latest updates: bit.ly/42vhqc9
Our leaders will participate in the William Blair 45th Annual Growth Stock Conference 2025 today June 3 at 10:20 a.m. ET. Tune in to our fireside chat to hear our latest updates: ir.ptcbio.com/events-present…
Our leaders will be participating in the RBC Capital Markets Global Healthcare Conference 2025 today, May 20 at 2:05 p.m. ET. Tune in to our fireside chat to hear our latest updates: bit.ly/42vhqc9
We invite you to hear from PTC's leaders at the Bank of America Securities 2025 Health Care Conference today, May 13. Tune in to our live webcast at 1 p.m. ET (10 a.m. PT) to learn more about the company's latest updates: bit.ly/42vhqc9
Today we announced our first quarter 2025 earnings, highlighting a promising start thanks to strong revenue performance and a steady progression of global launch activities following the recent positive CHMP opinion for our potential PKU treatment. bit.ly/3RROoQc
Today, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion on PTC's treatment option Sephience (sepiapterin) for adults and children with phenylketonuria (PKU). Learn more: bit.ly/4jUzQex
PTC's leaders will present at four upcoming investor conferences this May and June. Learn more: bit.ly/3S4PB6B
Today we announced that our leaders will report the company's first-quarter 2025 financial results and provide an update on the company's business and outlook on Tuesday, May 6 at 4:30 p.m. ET. Learn more: bit.ly/42wwiYD
We are pioneering innovative treatments for patients around the world and are proud to share that our #genetherapy for AADC deficiency (#AADCd) has received approval in Hong Kong.
Our teams were thrilled to connect with the #Friedreichsataxia (FA) community at the 2025 #MDAConference and share data from our #FA program. We're proud to be developing a potential treatment for children and adults living with this rare disorder.
It's been a productive week for us at #ACMGmtg25. We presented new data from our sepiapterin Phase 3 APHENITY trial and open-label extension study demonstrating the potential to treat children and adults living with #PKU. Learn more: bit.ly/4bEWWCV
We're excited to be onsite at this year's #ACMGMtg25 in Los Angeles to to share data on our #PKU and #AADCd programs.
We're honored to be named one of Gallup's 2025 Exceptional Workplace Award winners for Strengths Development for the fourth time! Learn more: bit.ly/4hvd239

ClearPointNeuro @ClearPointNeuro
2K Followers 180 Following Global device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine. #CED #CLPT #Nsgy #DBS #LITT #GeneTherapy #SmartFlow
BattlinBilly @SRQForever
965 Followers 904 Following
BullishDoctor @NeuroStockDoc
6K Followers 1K Following MD. Neurologist. Journaling investing journey. Micro through Large Caps.
Jacqueline Keller @canuck1973
724 Followers 759 Following Chief Marketing Officer at Neurochase - the premier next generation platform for drug delivery to the brain #CED
CheckRare @CheckRare
3K Followers 2K Following Leading publisher and learning platform focused on rare diseases. Rare Diseases Are Our Focus, Expertise, and Passion.
AnnemiekeAartsma-Rus @oligogirl
3K Followers 456 Following Translating science from bench to bedside and from jargon to lay language
Beacon for Rare Disea... @RareBeacon
12K Followers 10K Following Beacon is a UK-based charity that is building a united rare disease community with patient groups at its heart. Previously known as Findacure.
Logos LP @Logos_LP
11K Followers 1K Following Interdisciplinary Value Investing. Tweets not investment advice or solicitations/offers to buy/sell securities. Distribution list: https://t.co/myEluBjaAi
RARE Revolution Magaz... @RareRevolutionM
12K Followers 7K Following Digital magazine giving a voice to those affected by rare conditions and the charities that support them. Contact us: [email protected]
TREAT-NMD® @TREAT_NMD
4K Followers 542 Following Advancing diagnosis, care and treatment for those living with neuromuscular diseases around the world.
World Muscle Society @WorldMuscleSoc
2K Followers 627 Following The WMS is a global, multidisciplinary community committed to advancing the science of neuromuscular disorders. Join us in Vienna for #WMS2025.
Effie Parks @OnceUponAGene
6K Followers 4K Following Rare Disease Advocate | Award Winning Podcaster | Speaker | Captain Connection | RareMama to my sweet, Ford, who lives with #CTNNB1 🦓
EveryLife Foundation @EveryLifeOrg
7K Followers 3K Following Nonprofit org. dedicated to advancing the development of treatment & diagnostic opportunities for rare disease patients through science-driven public policy.
Edgewise Therapeutics @EdgewiseTx
505 Followers 47 Following On a mission to improve the lives of people facing serious muscle disease.
Rare Patient Voice @rarepatientvoic
2K Followers 3K Following We help clients find rare & non-rare disease patients & caregivers for research studies, & connect patients & caregivers with paid research opportunities.
HDYO @HDYOFeed
2K Followers 725 Following An international non-profit organisation supporting, educating & empowering young people, up to 35 years, impacted by Huntington’s Disease around the world.
Patient Worthy @PatientWorthy
8K Followers 7K Following We're a resource for engaging, informative content and rare patient news, well done.
Huntington's Disease ... @HDA_tweeting
4K Followers 2K Following Huntington's Disease Association is a registered charity that supports people affected by #HuntingtonsDisease (HD) in England and Wales. #MindfulOfHuntingtons
CureDuchenne @CureDuchenne
6K Followers 582 Following We are committed to improving the lives of those affected by Duchenne through accelerating research, improving care and empowering the community.
FEDER | Enfermedades ... @FEDER_ONG
39K Followers 8K Following 👉🏻 Somos la esperanza de 3 millones de personas con #enfermedadesraras 🍀 Representamos a 442 organizaciones de pacientes y a 1.546 patologías poco frecuentes
Xawe @Xawe798108
8 Followers 368 Following
EMJ GOLD @EMJ_GOLD
2K Followers 1K Following Join our mailing list for FREE to receive the latest pharma news, views and analysis direct to your inbox: https://t.co/XzaDMFdtXF
MAAV @MARAV1992
0 Followers 84 Following
Julie Nouet @JulieNouet
8 Followers 99 Following French American plant based foodie, mol biology scientist
jaideb bakshi @jaideb
8 Followers 59 Following
Rahmi Arslan @rahmi_arslan1
0 Followers 8 Following
Linda Lee @lindaswilleylee
31 Followers 146 Following retired Lumberton Public School District --- 30 Long Years
Yingying Liu @helenlen_liu
2 Followers 53 Following
Tsipi.B @TsipiB
55 Followers 510 Following אמא , עורכת דין, קצינה במיל. , אוהבת ללמוד , מתעניינת במדע אקטואליה וכו
Raveendra @CaptRav73662
28 Followers 284 Following
長谷山 正樹 @manhattan0131
6 Followers 71 Following
Abdul Shaikh @ShaikhIgnited
2 Followers 15 Following
Amy Hillegas @AmyHillegas
284 Followers 1K Following
Inside Drug Discovery @insidedrugdisc
3 Followers 24 Following This account announces new video releases for https://t.co/SSFPDEOrFR. The account is managed by Erland Stevens.
Natasha Fox (Cotter) @foxcot
712 Followers 2K Following Mum of beautiful brave girl born with PKU. Senior leader. NPQH. Swim mum and swim coach. Passionate about teachers being valued. All views my own.
Lic.Agush11 @agustinadducci
330 Followers 543 Following
Mgt Lappin @MargaretLappin
248 Followers 511 Following Mum to an amazing 23 year old who is my daughter, my best friend and my star 🌟 #true love
Sharon Buckley @SharonBuckley
975 Followers 520 Following
matei alexandru @sasamatei
25 Followers 87 Following
Frieda wachsmann @WachsmannFrieda
1 Followers 18 Following
Amazing M ( Real Me) @AmazingM18
43 Followers 1K Following Be good, Do good ,Spread Love, Happinesses, and Positivity. Contribute to World Peace ☮️, Cancer Research. #Medcomms, Clinical Trials
Helena Siobhan @HelenaSiobhan
3 Followers 43 Following
Rosenhal @Rosenhal_233
6 Followers 284 Following
Tian Mack @QweAdc61334
16 Followers 921 Following OEM optical Component Manufacturer!#laser#crystals #semiconductors #prsims #sapphire #dome#metallization #Coating #germanium (Ge)#fibreoptics
Hatt •|||||||• @madhatter_202
437 Followers 5K Following
Hunter @Hunter140033
81 Followers 630 Following
trickyyy @papatops9
14 Followers 155 Following
((Andrew Natenshon)) @Natenshon
239 Followers 4K Following Working to get treatments out for devastating rare diseases Portfolio Lead @Moderna_tx Tweets are my own.
Transit @TrustTransit
2K Followers 3K Following Cinema, photography, technology, online privacy and global health. I share my take and reviews on these topics.
Kate Learoyd @PKUFamily
1K Followers 1K Following Mum, charity trustee, ask awkward questions, trying to cope with this icky platform: support @NSPKU for better future for #RareDisease #pku, tweeting own views
Rachel Brannick 🇵�... @rachel_brannick
191 Followers 1K Following PhD Researcher in Law and Criminology @maynoothlaw | Researching the impact of technology on conflict-related sexual violence | PKU mother and advocate
Sr simpático @Don_Simpaticon
35 Followers 180 Following
ノビ @KIupJwwZQJmctps
16 Followers 64 Following 異業種から転職して現在、コントラクトMR→外資製薬メーカーMR。皆様のつぶやきから活力貰ってます!無言フォローすみません。
Jacksyn @jjacksynn
1 Followers 4 Following
梁铭军 @9S3n4tjLw740868
0 Followers 3 Following
HereForScience @HereForGenetics
4 Followers 125 Following
ADP .. @adp0424
1 Followers 930 Following
梁铭军 @zzfIvslRClfRdpD
0 Followers 11 Following
Swapnil Mudhalwadkar @iam_swapam
1 Followers 39 Following
Navreet Kaur @navreetdoad
1 Followers 4 Following
CALL ME SNOWBALL! @yagogarita
145 Followers 484 Following Chaos is a ladder 🏳️🌈 Si esperas el momento oportuno, era ese
Farida ElGamal @ElgamalFarida
0 Followers 13 Following
Shamim Dadvar @shamimdadvar
205 Followers 861 Following Life science venture capital @HealthcapVC | MD, PhD @karolinskainst @ruaslab | Views are my own
RANGAN @RANGANMALLIK
131 Followers 207 Following
Neurochase - Next Gen... @neurochase
76 Followers 103 Following Global platform company focused exclusively on innovative devices & consulting services for CNS drug delivery #CED #genetherapy #celltherapy #intraparenchymal
ClearPointNeuro @ClearPointNeuro
2K Followers 180 Following Global device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine. #CED #CLPT #Nsgy #DBS #LITT #GeneTherapy #SmartFlow
Jacqueline Keller @canuck1973
724 Followers 759 Following Chief Marketing Officer at Neurochase - the premier next generation platform for drug delivery to the brain #CED
CheckRare @CheckRare
3K Followers 2K Following Leading publisher and learning platform focused on rare diseases. Rare Diseases Are Our Focus, Expertise, and Passion.
AnnemiekeAartsma-Rus @oligogirl
3K Followers 456 Following Translating science from bench to bedside and from jargon to lay language
RARE Revolution Magaz... @RareRevolutionM
12K Followers 7K Following Digital magazine giving a voice to those affected by rare conditions and the charities that support them. Contact us: [email protected]
Global Genes @GlobalGenes
29K Followers 6K Following Empowering the Next Generation Rare Disease Advocate. Merged with RARE-X Dec. 2022. #CareAboutRare
TREAT-NMD® @TREAT_NMD
4K Followers 542 Following Advancing diagnosis, care and treatment for those living with neuromuscular diseases around the world.
Parent Project Muscul... @ParentProjectMD
7K Followers 991 Following Let's advance the research evolution. #EndDuchenne
World Muscle Society @WorldMuscleSoc
2K Followers 627 Following The WMS is a global, multidisciplinary community committed to advancing the science of neuromuscular disorders. Join us in Vienna for #WMS2025.
Rare Disease Day @rarediseaseday
42K Followers 3K Following 29 February 2024 is Rare Disease Day. Raising awareness for patients, families and carers around the world that are affected by rare diseases. #RareDiseaseDay
Effie Parks @OnceUponAGene
6K Followers 4K Following Rare Disease Advocate | Award Winning Podcaster | Speaker | Captain Connection | RareMama to my sweet, Ford, who lives with #CTNNB1 🦓
EURORDIS-Rare Disease... @eurordis
32K Followers 1K Following An alliance of over 1,000 patient organisations working across borders and diseases to improve the lives of all people living with rare diseases.
EveryLife Foundation @EveryLifeOrg
7K Followers 3K Following Nonprofit org. dedicated to advancing the development of treatment & diagnostic opportunities for rare disease patients through science-driven public policy.
HDYO @HDYOFeed
2K Followers 725 Following An international non-profit organisation supporting, educating & empowering young people, up to 35 years, impacted by Huntington’s Disease around the world.
Patient Worthy @PatientWorthy
8K Followers 7K Following We're a resource for engaging, informative content and rare patient news, well done.
Huntington's Disease ... @HDA_tweeting
4K Followers 2K Following Huntington's Disease Association is a registered charity that supports people affected by #HuntingtonsDisease (HD) in England and Wales. #MindfulOfHuntingtons
CureDuchenne @CureDuchenne
6K Followers 582 Following We are committed to improving the lives of those affected by Duchenne through accelerating research, improving care and empowering the community.
Duchenne UK @DuchenneUK
5K Followers 922 Following Duchenne UK has one clear aim – to end Duchenne, a devastating muscle-wasting disease. We are the leading Duchenne muscular dystrophy charity in the UK.
HDSA @HDSA
6K Followers 1K Following The Huntington’s Disease Society of America is the premier nonprofit organization dedicated to improving the lives of everyone affected by Huntington’s disease.
Muscular Dystrophy UK @MDUK_News
17K Followers 3K Following We connect a community of over 110,000 people living with muscle wasting and weakening conditions. Together we are stronger. Join us. Our #MusclesMatter.
HDBuzz @HDBuzzFeed
6K Followers 2K Following Huntington’s disease research news. In plain language. Written by scientists. Editors @labscribbles @rungenado
Huntington's Disease ... @HDAI_ie
3K Followers 5K Following HDAI RCN 20025656. Sharing news on Huntingtons Disease /connecting to the international HD community. RTs not endorsements. https://t.co/z0BAD0VUeS https://t.co/pPoXkLELx2
@HDAwareness. @HDAwareness1
118 Followers 287 Following Awareness leads to great things; Please Get involved what's left of my Family Thanks You. #huntingtondisease #juvenilehuntingtonsdisease #huntingtonsdisease
Madrigal Pharmaceutic... @MadrigalPharma
2K Followers 359 Following Madrigal is focused on delivering novel therapeutics for MASH, a serious liver disease.
Lauren Holder @laurencurehd
567 Followers 495 Following HD advocate, #rarediseases advocate, Producer of Help4HD Live, caregiver, mom, and much more!
Lisa's Legacy for ALS @LisaLegacy4ALS
2K Followers 874 Following Lisa Stockman Mauriello fought for Expanded Access to an ALS treatment. Lisa passed away 8.4.21. Honor Lisa’s legacy by advocating for ALS.
Screen4Care @Screen4Care
329 Followers 118 Following Accelerating Diagnosis for Rare Disease Patients Through Genetic Newborn Screening and Artificial Intelligence
Two Smiles One Hope @2Smiles1Hope
292 Followers 365 Following Team Two Smiles * Racing to END Duchenne Muscular Dystrophy
DUCHENNE ECUADOR F-A-... @FunArielChichoG
137 Followers 204 Following Somos una fundación sin fines de lucro enfocados en niñas, niños y adolescentes con discapacidad física y enfermedades raras. Otorgandoles CALIDAD DE VIDA.
Leiomyosarcoma Suppor... @2LMSdr
225 Followers 121 Following Leiomyosarcoma Support & Direct Research (LMSDR) raises awareness and funds for leiomyosarcoma research. We offer PATIENT EDUCATION, ADVOCACY & SUPPORT.
ESGCT @ESGCT
4K Followers 606 Following Official Twitter account of the European Society of Gene & Cell Therapy.
HuntingtonStudyGroup @HuntingtonSG
2K Followers 486 Following Accelerating treatments that make a difference for Huntington's disease.
Tyler Hernandez @therealtastyt
523 Followers 620 Following Florida CPA - Jesuit '14 UT '17 & ‘19 MACC - Team USA Power Wheelchair Soccer -Barstool Athlete- My tweets are my own.
NEALS Consortium @NEALSConsortium
3K Followers 97 Following The NEALS Consortium is an international, independent, non-profit group of researchers who collaboratively conduct clinical research in ALS
ALS TDI @ALSTDI
10K Followers 6K Following The most comprehensive lab focused on ALS/Lou Gehrig's Disease. We are the Drug Discovery Engine discovering & inventing effective treatments for ALS. #EndALS
Dean Williams @dwecintouch
4K Followers 4K Following Executive Coach and Chair of The Wheelchair Football Association
Jon Bolding @jbolding8
1K Followers 1K Following England Powerchair Football Captain 🏆 European Champion 🥇x2 🗣️ Speaker & Influencer in Sports 💪 Championing Inclusivity
2026 FIPFA Powerchair... @2023FIPFAPFWC
210 Followers 16 Following The 2026 FIPFA Powerchair Football World Cup will be held in Buenos Aires, Argentina, in September 2026.
Chris @CGordon_90
880 Followers 1K Following West Bromwich Albion and England Powerchair footballer. Working with Power Soccer Shop to make the Strike Force accessible in England!
Adrienne Frumberg @AdrienneFrum
37 Followers 170 Following An independent educational consultant/college counselor for high school students and young adults working to reach their goals.
HD Reach @HDReach
1K Followers 80 Following HD Reach is a nonprofit organization dedicated to providing access to healthcare, education, and social assistance for those affected by Huntington’s disease.
Cambridge Healthtech ... @CHI_Healthtech
8K Followers 2K Following CHI is the preeminent life science network for leading researchers and business experts from top pharmaceutical, biotech and academic organizations.
Alexa Tinsley @alexa_tinsley
11 Followers 11 Following Director of Community Engagement at Jett Foundation
Tuca Figueira @TucaFigueira
205 Followers 302 Following Relações Públicas de longa data. Apaixonada por saúde e gestão de crises.
MuscularDystrophyIRE @MDI_Ireland
2K Followers 909 Following Providing support services to people with neuromuscular conditions and their families and supports quality research.
Life Sciences Industr... @LifeSciencesIRL
62 Followers 61 Following The Life Science Industry Awards recognise and celebrate excellence in the Irish life science industry. #LifeSciencesIRL
Genentech @genentech
125K Followers 227 Following Official Twitter for Genentech. See our community guidelines here: https://t.co/yas51FPhKU
Lynnette KhalfaniCox @themoneycoach
17K Followers 3K Following Personal finance expert, speaker, author of Bounce Back: The Ultimate Guide to Financial Resilience. Seen on Oprah, Today, Dr. Phil, CNN, MSNBC, NewsNation.
The Galien Foundation @GalienFdn
2K Followers 2K Following A global foundation that fosters the open exchange of ideas to drive scientific #innovation in medicine. #PrixGalien.
World Orphan Drug Con... @OrphanConf
5K Followers 870 Following Get regular updates on #WorldOrphanUSA! June 9-11, 2026 | Boston Convention & Exhibition Center
Everyday is #HDAwaren... @AwarenessHD
2K Followers 5K Following Awareness leads to great things; Please Get involved what's left of my Family Thanks You. #huntingtondisease #juvenilehuntingtonsdisease #huntingtonsdisease
Rafael @_RafaelSierra
7 Followers 15 Following Medical Affairs Leader for Interested in #RareDiseases #Neurology #Liver #Hepatology #Bone
Rareminds @RaremindsUK
945 Followers 2K Following Specialised mental health and wellbeing services for the rare disease community. https://t.co/eVmeTw0aah…
CFO Intelligence @CfoIntelligence
27 Followers 8 Following CFO Intelligence drives relationships, insights & growth for CFOs and senior finance executives at enterprise and middle market companies
SPAGN @sarcomapatients
2K Followers 558 Following Sarcoma Patient Advocacy Global Network (SPAGN) is the global network of Sarcoma, GIST and Desmoid Patient Advocacy Groups
Lou Gehrig Day Commit... @LG4Day
3K Followers 909 Following Welcome to the Twitter home of Lou Gehrig Day committee, an annual MLB tradition on June 2, starting in 2021, to honor him and raise awareness for ALS!
Jennifer Roan @jenniferroan
28 Followers 479 Following
Patrice Thornton @PThornton76
171 Followers 949 Following VP HR | YNWA | 🐕 🐾🍷| Views expressed are my own
Elijah Stacy @Elijahjstacy
601 Followers 382 Following Author, Public Speaker, Biotech Consultant for 5 companies, Investor and Founder of Destroy Duchenne. Media/bookings: [email protected]. My book 👇
Odalys Martinez @OdalysTMartinez
42 Followers 81 Following
Duchenne Data Foundat... @DuchenneDF
778 Followers 434 Following Bringing data to life to improve the world of dystrophinopathies
FIPFA @OfficialFIPFA
1K Followers 156 Following Official tweets from the Fédération Internationale de Powerchair Football Association (FIPFA), the governing body of Powerchair Football worldwide 🌏
Suzie Siegel @SuzieSiegel
2K Followers 1K Following Newspaper reporter & editor b4 metastatic leiomyosarcoma. Master's in women's studies. Board member emerita of Sarcoma Alliance. urostomy colostomy
The WFA @The_WFA
3K Followers 665 Following Official account of The Wheelchair Football Association (WFA). Bringing you the latest news, updates & insights on Powerchair Football across England.