A continuation of Firefly Fund co-founder Pam Andrews' discussion with NPC moms, highlighting the benefits of Adrabetadex. The moms share their fears about a future without the medication, the popular topic of risk vs. benefit to administer the medication, as well as thoughts about adding additional therapies to treat NPC children and give them the best chance to live healthy lives.

Firefly Fund co-founder Pam Andrews has a heartfelt and candid conversation with nine other NPC moms from around the world regarding recent news of the discontinuation of the medication Adrabetadex. This episode gives a glimpse into the difficult circumstances NPC families are facing, as well as their personal concerns and fears about their children's futures.

Firefly Fund founders Pam and Chris Andrews chat with the Mallinckrodt team. Dr. Steve Romano, Elissa Cote, and Sheila Talafous share their journies to joining the Mallinckrodt team, how rare disease has impacted them, and what is coming up with the study of adrabetadex.

Firefly Fund founders Pam and Chris Andrews celebrate NPC Awareness Month by having a conversation with National Niemann-Pick Disease Foundation, Inc., Executive Director Joslyn Crowe, MSW, MA, and NNPDF Board Chair Justin Hopkin, MD. They discuss Joslyn & Justin's journeys to NNPDF, the resources NNPDF provides, and what is on the horizon.

This month's episode of Firefly Chat focuses on Newborn Screening Awareness Month. The podcast was repurposed from a discussion at the recent World Orphan Drug Congress, featuring speakers Firefly Fund Executive Director and Co-founder Pam Crowley Andrews; Pat Furlong, with Parent Project Muscular Dystrophy; Emily Fields of Bluebird Bio; Dean Suhr, of the MLD Foundation; and Dr. Melissa Wasserstein, from Montefiore Medical Center. The panel was moderated by Firefly Fund friend and consultant to Firefly's Newborn Screening Initiative, Allison May Rosen.

Tune in for the second installation of Firefly Fund Co-Founders Pam & Chris Andrews' conversation with Thomas Kirkegaard Jensen Ph.D., Co-Founder and Chief Scientific Officer and Kim Stratton, CEO of Orphazyme, a biopharmaceutical company that translates scientific discoveries into late-stage clinical development programs impacting lives of patients with orphan diseases and their families.

Firefly Fund Co-Founders Pam & Chris Andrews enjoyed their recent Firefly Chat with Thomas Kirkegaard Jensen Ph.D., Co-Founder and Chief Scientific Officer of Orphazyme and Kim Stratton, CEO of Orphazyme. We know you will like getting to know Thomas and Kim as much as we did and that you will also appreciate hearing about the history of Orphazyme and the development of Arimoclomol, a novel treatment for Niemann Pick Type C disease that has completed a Phase three clinical trial and recently filed a New Drug Application (NDA) with the USFDA. Orphazyme is a biopharmaceutical company that translates scientific discoveries into late-stage clinical development programs impacting lives of patients with orphan diseases and their families.

Join Firefly Fund founders Pam and Chris Andrews as they speak about the future of rare disease treatment with John Crowley, Chairman and CEO of Amicus Therapeutics — a global, patient-dedicated biotech focused on discovering, developing, and delivering high-quality medicines for people living with rare metabolic diseases. Prior to becoming the head of Amicus, Crowley co-founded Novazyme Pharmaceuticals, which was later acquired by Genzyme Corporation. Crowley also served as an intelligence officer in the U.S. Navy Reserve. He and his family were the inspiration for the movie Extraordinary Measures starring Harrison Ford and Brendan Fraser in 2010.

Join Firefly Fund founders Pam and Chris Andrews as they share about their own personal rare disease journey that led them to the creation of Firefly Fund, as well as what you can expect from the monthly Firefly Chat. The purpose of Firefly Chat is to provide a platform for all NPC Community and industry stakeholders to share and air their views to bring together the whole NPC Community, while also shining a light on the collective dedication and commitment to NPC patients.