Tagged with Paediatrics

USA Matt Heck, founder and CEO of Sentynl Therapeutics, outlines the company’s acquisition-led rare disease strategy following its integration into Zydus Group in 2017. He discusses building a focused portfolio of life-extending paediatric therapies, leveraging orphan drug incentives, prioritising early diagnosis, expanding internationally, and partnering closely with patient foundations to maximise…

USA Dr Carlos Prada, Director of the Edwards Family Division of Genetics and Rare Diseases at Ann & Robert H. Lurie Children’s Hospital of Chicago, discusses how earlier diagnosis, coordinated care pathways, and emerging therapeutic modalities are reshaping outcomes for children and families. From newborn screening and multidisciplinary follow-up to the…

USA Dr Jerry Vockley, Director of the Center for Rare Disease Therapy and Chief of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, offers a look at the evolving landscape of rare disease care across early diagnosis, newborn screening, natural history research, clinical trial design, and the persistent…

Hong Kong At Hong Kong Children’s Hospital (HKCH), a new chapter in rare disease medicine is unfolding. Under the leadership of Dr Luk Ho-ming, Chief of Service of the Department of Clinical Genetics, a multidisciplinary team is harnessing the power of genomics, data, and collaboration to bring answers to families who have…

Hong Kong Established in 2018 under the Hospital Authority, the Hong Kong Children’s Hospital (HKCH) has rapidly emerged as the city’s leading centre for complex paediatric and rare-disease care. Bringing together advanced diagnostics, pioneering research, and multidisciplinary expertise under one roof, HKCH represents a new model of integrated medicine in Asia, one…

UAE Dr Entesar Al Hammadi has held leadership positions in paediatric hospitals in the United Arab Emirates (UAE) for the past 23 years. She is currently heading up the Al Jalila Children’s Hospital’s academic affairs department as well as serving as its deputy medical director. Dr Al Hammadi is also the…

Austria Prof. Ruth Ladenstein, executive director of the Medicines for Children Research Organisation (OKIDS) in Austria, explains OKIDS’ drive to ensure drugs are paediatric specific by conducting child-specific clinical trials. Furthermore, she highlights the challenges associated with achieving a participating child’s consent and defines what must be done to gain governmental…