medhub.ptcbio.comLatest scientific data and expert insights | MEDhub Home - MEDhub

Open search field Neuromuscular Duchenne Muscular Dystrophy Disease overview Congress activities Publications Webinars Hereditary Transthyretin (hATTR) Amyloidosis Disease overview Congress activities Publications Webinars Central nervous system AADC Deficiency Disease overview Congress activities Publications Webinars Friedreich’s Ataxia Disease overview Congress activities Publications Webinars Huntington’s Disease Disease overview Congress activities Publications Webinars Amyotrophic lateral sclerosis (ALS) Disease overview Congress activities Publications Webinars Metabolism Familial Chylomicronemia Syndrome (FCS)​ Disease overview Congress activities Publications Webinars Phenylketonuria (PKU)​ Disease overview Congress activities Publications Webinars You are browsing the Global version of MEDHub. Click here to change. Search Search The latest scientific data and expert insights delivered to you Supporting healthcare professionals worldwide with best practice for the early and accurate diagnosis and management of rare diseases – providing you with the latest scientific data and expert insights on the diagnosis and management of a range of rare diseases across neuromuscular, central nervous system and other areas MED-ALL-CORP-2200029 | October 2023 Just added Top content Muscular Dystrophy Association (MDA) Annual ClinicalScientific Conference 07/03/2024 MDA 2024 poster: Improvement in upright stability subscale of mFARS with vatiquinone treatment in MOVE-FA: A... International Symposium on Paediatric Movement Disorders (ISPMD) 12/02/2024 ISPMD 2024 poster: Pharmacodynamics of eladocagene exuparvovec and safety of the SmartFlow magnetic resonance-compatible ventricular cannula... Muscular Dystrophy Association (MDA) Annual ClinicalScientific Conference 07/03/2024 MDA 2024 oral presentation: Safety and efficacy of vatiquinone treatment in Friedreich Ataxia patients from MOVE-FA:... Virology 04/10/2021 Coronavirus (COVID-19) Friedreich’s ataxia 07/03/2024 Muscular Dystrophy Association (MDA) Annual ClinicalScientific Conference Duchenne muscular dystrophy (DMD) 21/12/2022 Interactive case study: Oliver’s diagnostic journey Muscular Dystrophy Association (MDA) Annual ClinicalScientific Conference 07/03/2024 MDA 2024 poster: Ataluren delays clinically meaningful milestones of decline in 6MWD in patients with nmDMD... Aromatic L-amino acid decarboxylase (AADC) deficiency 24/01/2022 International Symposium on Paediatric Movement Disorders (ISPMD) Just added Muscular Dystrophy Association (MDA) Annual ClinicalScientific Conference 07/03/2024 MDA 2024 poster: Improvement in upright stability subscale of mFARS with vatiquinone treatment in MOVE-FA: A... Muscular Dystrophy Association (MDA) Annual ClinicalScientific Conference 07/03/2024 MDA 2024 oral presentation: Safety and efficacy of vatiquinone treatment in Friedreich Ataxia patients from MOVE-FA:... Friedreich’s ataxia 07/03/2024 Muscular Dystrophy Association (MDA) Annual ClinicalScientific Conference Muscular Dystrophy Association (MDA) Annual ClinicalScientific Conference 07/03/2024 MDA 2024 poster: Ataluren delays clinically meaningful milestones of decline in 6MWD in patients with nmDMD... Top content International Symposium on Paediatric Movement Disorders (ISPMD) 12/02/2024 ISPMD 2024 poster: Pharmacodynamics of eladocagene exuparvovec and safety of the SmartFlow magnetic resonance-compatible ventricular cannula... Virology 04/10/2021 Coronavirus (COVID-19) Duchenne muscular dystrophy (DMD) 21/12/2022 Interactive case study: Oliver’s diagnostic journey Aromatic L-amino acid decarboxylase (AADC) deficiency 24/01/2022 International Symposium on Paediatric Movement Disorders (ISPMD) Central Nervous System View content Metabolism View content Neuromuscular View content Sign in or register to access exclusive content on this site SIGN IN REGISTER Authenticating... Contact Us MED-ALL-CORP-2200029 | October 2023 This website is organized and funded by PTC Therapeutics for healthcare professionals only. For medical inquiries get in touch at MedInfo@ptcbio.com This website may include scientific information about investigational and unlicensed products not approved for use within your country. Please refer to local regulatory guidance for country-specific information © 2023 PTC Therapeutics. All rights reserved TermsConditions Privacy Statement Cookies Statement Welcome to MEDhub A website for healthcare professionals, provided by PTC Therapeutics This educational website provides you with the latest scientific data and expert insights on the diagnosis and management of a range of rare diseases across the neuromuscular system, central nervous system, and other areas Based on your location we think the relevant MEDhub site for you is: United States if this is incorrect, please select the appropriate local market for you: Afghanistan Albania Algeria American Samoa Andorra Angola Anguilla Antarctica Antigua and Barbuda Argentina Armenia Aruba Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bermuda Bhutan Bolivia Bosnia and Herzegovina Botswana Bouvet Island Brazil British Indian Ocean Territory Brunei Darussalam Bulgaria Burkina Faso Burundi Cambodia Cameroon Canada Cape Verde Cayman Islands Central African Republic Chad Chile China Christmas Island Cocos (Keeling) Islands Colombia Comoros Congo Congo, the Democratic Republic of the Cook Islands Costa Rica Cote D'Ivoire Croatia Cuba Cyprus Czech Republic Denmark Djibouti Dominica Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Ethiopia Falkland Islands (Malvinas) Faroe Islands Fiji Finland France French Guiana French Polynesia French Southern Territories Gabon Gambia Georgia Germany Ghana Gibraltar Greece Greenland Grenada Guadeloupe Guam Guatemala Guinea Guinea-Bissau Guyana Haiti Heard Island and Mcdonald Islands Holy See (Vatican City State) Honduras Hong Kong Hungary Iceland India Indonesia Iran, Islamic Republic of Iraq Ireland Israel Italy Jamaica Japan Jordan Kazakhstan Kenya Kiribati Korea, Democratic People's Republic of Korea, Republic of Kuwait Kyrgyzstan Lao People's Democratic Republic Latvia Lebanon Lesotho Liberia Libyan Arab Jamahiriya Liechtenstein Lithuania Luxembourg Macao Macedonia, the Former Yugoslav Republic of Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Martinique Mauritania Mauritius Mayotte Mexico Micronesia, Federated States of Moldova, Republic of Monaco Mongolia Montserrat Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands Netherlands Antilles New Caledonia New Zealand Nicaragua Niger Nigeria Niue Norfolk Island Northern Mariana Islands Norway Oman Pakistan Palau Palestinian Territory, Occupied Panama Papua New Guinea Paraguay Peru Philippines Pitcairn Poland Portugal Puerto Rico Qatar Reunion Romania Russian Federation Rwanda Saint Helena Saint Kitts and Nevis Saint Lucia Saint Pierre and Miquelon Saint Vincent and the Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia and Montenegro Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Georgia and the South Sandwich Islands Spain Sri Lanka Sudan Suriname Svalbard and Jan Mayen Swaziland Sweden Switzerland Syrian Arab Republic Taiwan, Province of China Tajikistan Tanzania, United Republic of Thailand Timor-Leste Togo Tokelau Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Turks and Caicos Islands Tuvalu Uganda Ukraine United Arab Emirates United Kingdom United States United States Minor Outlying Islands Uruguay Uzbekistan Vanuatu Venezuela Viet Nam Virgin Islands, British Virgin Islands, U.s. Wallis and Futuna Western Sahara Yemen Zambia Zimbabwe I am a healthcare professional - Continue to website: medhub.ptcbio.com/en-us This site is not intended for members of the public, patients or their caregivers I am not...