LGBTQIA+ Affirming Prenatal Genetic Counseling

Join PhenoTips live on June 26th from 12 – 1 p.m EST, for the 29th installment of PhenoTips’ Speaker Series, “LGBTQIA+ Affirming Prenatal Genetic Counseling” LGBTQIA+ Affirming Prenatal Genetic Counseling […]
CAP/ACP Porphyria Genes Presentation and Annual General Meeting Registration

Join Dr. Aneal Khan to learn about the genetic basis of porphyria and the implications of new advancements in genetics for the diagnosis and treatment of rare diseases like porphyria!
Gene Therapy Webinar Series: Episode 10 – Genetic therapies and therapy developments for SMA

Jan Kirschner (Professor of Pediatric Neurology) has been joint coordinator of the German network for neuromuscular disorders MD-NET (www.md-net.org) since 2008. He has been involved in planning and conducting several […]
Gene Therapy Webinar Series – Episode 11: Safety issues in gene therapies and how to address them

Professor Francesco Muntoni is a Paediatric Neurologist with an interest in clinical, pathological and molecular aspects of neuromuscular disorders. He is the director of the Dubowitz Neuromuscular Centre, a leading […]
Gene Therapy Webinar Series: Episode 8 – Genetic therapies and therapy developments for ALS

Dr Vázquez Costa has a MD in Neurology (2012) and PhD in motor neuron diseases (2019). Since 2013 he is devoted to the care of motor neuron disease patients and […]
Gene Therapy Webinar Series: Episode 7 – Health technology assessment (HTA) of genetic therapies

Oriana Ciani is an Associate Professor of Practice in the Public Management and Policy, Health Economics, and HTA (Health Technology Assessment) Area at SDA Bocconi School of Management. Her collaboration […]
Gene Therapy Webinar Series: Episode 6 – Genetic therapies and therapy developments for muscular dystrophies (DMD, LGMD)

Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center (LUMC, the Netherlands). She played an important role […]
Gene Therapy Webinar Series – Episode 5: Genetic therapies and therapy developments for epileptic disorders

Dimitri Kullmann is a consultant neurologist at the National Hospital for Neurology and Neurosurgery, and professor of neurology at the UCL Queen Square Institute of Neurology. He trained in Oxford and […]
World Orphan Drug Congress 2024

The World Orphan Drug Congress is the largest, most established European meeting dedicated to orphan drugs. This year the conference will take place in Barcelona, Spain. From cell & gene […]
World Congress for Rare Skin Disorders

The objectives of the world congress for rare disorders are: To learn on diagnosis (including pathophysiology and disease classification) To develop a multidisciplinary approach and improve their skills to manage […]