ISCBH Webinars

A SERIES OF THREE ISCBH WEBINARS FOR 2025

This new series will cover the following topics:

Achondroplasia & Hypochondroplasia

Hypophosphatasia

Osteogenesis Imperfecta

For each of our webinars, we are excited to be addressing some of the issues that we all need answers to – What about the unmet needs of patients? What if there was no limit to funding?  Where have we been and where are we going?   

We’ll be looking into each topic with a futuristic lens… and welcoming plenty of engagement between our moderators and audience!  Together we can work to channel our future efforts to address those unmet needs.

The webinars will last one hour, starting with a 25 minute talk by our guest speaker, followed by a 35 minute Q&A and discussion session chaired by Professor Leanne Ward and Professor Erik Imel.

Webinar Series Convenors:

Erik Imel

Indianapolis, USA

Leanne Ward

Leanne Ward

Ottowa, Canada

'The Future of...' Webinar #2

THE FUTURE OF…

Hypophosphatasia

Dr Jill Simmons Nashville, USA
Tuesday 4 November 2025
14:00-15:00 CST, 15:00-16:00 EST, 20:00-21:00 GMT CHAIRS: Erik Imel & Leanne Ward

Dr Jill Simmons

Dr. Jill Simmons is a Professor of Pediatrics in the Ian M Burr Division of Pediatric Endocrinology and Diabetes at Vanderbilt University Medical Center. She is the founder and director of the Vanderbilt Program for Pediatric Metabolic Bone Disorders, the founder and chair of the Hazinski Society for Faculty Development, and the Director for Faculty Mentorship Programs in the Department of Pediatrics at Vanderbilt University.  She is a member of the scientific advisory board for Soft Bones as well as the Rare Bone Disease Alliance.

Dr. Simmons’ clinical and research interests include type 1 diabetes as well as pediatric metabolic bone diseases such as hypophosphatasia, osteogenesis imperfecta, osteopetrosis, hypophosphatemia, osteoporosis, and rickets. She has been a successful local principal investigator for multiple groundbreaking clinical trials in metabolic bone disease, including asfotase alpha in infants with severe hypophosphatasia, romozosumab and setrusumab in pediatric patients with osteogenesis imperfecta, and burosumab in pediatric patients with hypophosphatemic rickets.  She is also an active investigator in several international longitudinal observational registries and multiple ongoing clinical trials.

Dr. Simmons has published more than 80 manuscripts in peer reviewed journals, including but not limited to the New England Journal of Medicine, Lancet, Journal of the American Medical Association, Journal of Pediatrics, and Diabetes Care.  She serves as a regular reviewer for journals in her field and regularly presents her research at national and international meetings.

In addition to her research, she is an active clinician and educator.  She spends the majority of her time with patients as well as educating pediatric residents and endocrinology fellows to better care for children with endocrine diseases. She has won the highest patient experience award at Vanderbilt University Medical Center every year since its inception in 2017.

Previous Webinars

The recording of the Winter Webinar is available for viewing for ISCBH Members and can be found on the Members section of the website.

Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our Rare Disease division is focused on generating scientific and technological breakthroughs for people living with a rare disease via the discovery and development of integrated therapeutic solutions and novel indications of established medicines in rare and ultra-rare blood, endocrine and renal disorders. Novo Nordisk employs about 50,800 people in 80 countries out of which 3.300 within rare disease.

Kyowa Kirin strives to create and deliver novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company with a heritage of 70+ years, we apply cutting-edge science including an expertise in antibody research and engineering, to address the needs of patients and society across multiple therapeutic areas including Nephrology, Oncology, Immunology/Allergy and Neurology. Across four regions – Japan, Asia Pacific, North America and EMEA/International – we focus on our purpose, to make people smile, and are united by our shared values of commitment to life, teamwork/Wa*, innovation and integrity.

*Harmony and loop among people.

Founded in 2007, Ascendis Pharma is applying its innovative TransCon™ technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by our core values of patients, science and passion, we use our TransCon technologies to fulfill our mission of developing new and potentially best-in-class therapies that address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States.

Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases driven by pathologic mineralization and intimal proliferation which impact the vasculature, soft tissue and skeleton. We are initially focused on developing an enzyme therapy to treat ENPP1 and ABCC6 Deficiencies, rare genetic diseases associated with significant morbidity, mortality, and unmet medical need.

BioMarin is a world leader in developing and commercializing innovative therapies for rare diseases driven by genetic causes. With a 20-year history, BioMarin remains steadfast to its original mission—to bring new treatments to market that will make a big impact on small patient populations. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options, and are usually ignored. Visit www.biomarin.com to learn more.

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.