“So Chocolate Bar” – 6 years later and one boy’s idea to help his friend with GSD1b is leading to scientific breakthroughs.
Ultragenyx and Arcturus announce expansion of their research collaboration to develop gene therapies for rare diseases.
Dr Guiseppe Ronzitti, a group leader at Généthon, confirmed to speak on gene therapy developments for GSDs at our 2019 conference.
Manchester Metropolitan University is investigating how bladder problems affect quality of life in neuromuscular conditions, including Pompe and McArdle’s.
”Pompe Disease News“ reports on a new genetic variant which may help explain the variability of symptom onset in Pompe disease.