Tagged with Paediatric

Matt Heck – Founder & CEO, Sentynl Therapeutics

USA Matt Heck – Founder & CEO, Sentynl Therapeutics

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…

France’s Patient Advocacy Power

France France’s Patient Advocacy Power

France France’s patient advocacy network is one of Europe’s most well-developed and formalised. Patient advocacy groups – including the three featured below – fill in research, care, and support gaps left by private industry and the state while elevating the concerns of the most important people within healthcare: patients and their…

Carlos Prada- Division Head, Edwards Family Division of Genetics and Rare Diseases, Ann & Robert H Lurie Children’s Hospital of Chicago

USA Carlos Prada- Division Head, Edwards Family Division of Genetics and Rare Diseases, Ann & Robert H Lurie Children’s Hospital of Chicago

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…

Jerry Vockley – Director of the Center for Rare Disease Therapy and Chief of the Division of Medical Genetics, UPMC Children’s Hospital of Pittsburgh

USA Jerry Vockley – Director of the Center for Rare Disease Therapy and Chief of the Division of Medical Genetics, UPMC Children’s Hospital of Pittsburgh

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…

LUK Ho-ming – Chief of Service (Clinical Genetics), Hong Kong Children’s Hospital

Hong Kong LUK Ho-ming – Chief of Service (Clinical Genetics), Hong Kong Children’s Hospital

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…

Chloe Mak – Consultant (Pathology) & Fung Cheuk-wing – Consultant (Paediatrics & Adolescent Medicine), Hong Kong Children’s Hospital

Hong Kong Chloe Mak – Consultant (Pathology) & Fung Cheuk-wing – Consultant (Paediatrics & Adolescent Medicine), Hong Kong Children’s Hospital

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…

Entesar Al Hammadi – Director of Academic Affairs & Deputy Medical Director, Al Jalila Children’s Specialty Hospital; President, Emirates Pediatrics & Neonatal Society

UAE Entesar Al Hammadi – Director of Academic Affairs & Deputy Medical Director, Al Jalila Children’s Specialty Hospital; President, Emirates Pediatrics & Neonatal Society

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…

Amel El Beshlawy – Professor of Paediatric Haematology, Paediatric Hospital of Cairo University

Egypt Amel El Beshlawy – Professor of Paediatric Haematology, Paediatric Hospital of Cairo University

Egypt Dr Amel El Beshlawy, founder of Egyptian Thalassemia Association (ETA) and Professor of Pediatric Hematology at the Pediatric Hospital of Cairo University, is considered the Middle East’s leading authority on Thalassemia, having made an enduring contribution not only to improving the survival rate of thalassemia patients in her clinical practice,…

Navin Pinto – Attending Physician, Seattle Children’s Hospital; Associate Professor of Paediatrics, University of Washington School of Medicine

USA Navin Pinto – Attending Physician, Seattle Children’s Hospital; Associate Professor of Paediatrics, University of Washington School of Medicine

USA Paediatric oncologist Navin Pinto, MD outlines the current use and potential of CAR-T therapies in his field, including the manufacturing challenges that still need to be addressed. Dr Pinto also explains how the work of his CAR-T team at the University of Washington School of Medicine could provide proof of…

Sam Volchenboum – Associate Professor of Pediatrics, Dean of Master’s Education, Associate Chief, Research Informatics Officer Associate Director, Institute for Translational Medicine, University of Chicago

USA Sam Volchenboum – Associate Professor of Pediatrics, Dean of Master’s Education, Associate Chief, Research Informatics Officer Associate Director, Institute for Translational Medicine, University of Chicago

USA Dr Sam Volchenboum outlines the progress that has been made in the paediatric oncology field in recent years and his hopes for its future. Dr Volchenboum also explains the important role that data will play in the new era of targeted and personalised therapies and the impact of the work…

Interview: Ruth Ladenstein – Executive Director, OKIDS, Austria

Austria Interview: Ruth Ladenstein – Executive Director, OKIDS, Austria

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…

Interview: Kuntal Baveja – Country Head & President, Sandoz Philippines

Philippines Interview: Kuntal Baveja – Country Head & President, Sandoz Philippines

Philippines Kuntal Baveja, country head and president of Sandoz Philippines Corporation discusses the company’s expanding portfolio in the country and how the booming generic industry over the past years has helped to expand access to affordable medications to the Filipinos, still pointing out that there is much room for expansion in…