Firefly Chat

Firefly Chat Episode 7 (Part 2): NPC Moms React to Adrabetadex News

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 Chat Episode 7(Part 1): NPC Moms React to Adrabetadex News

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 Chat Episode 6: Mallinckrodt Pharmaceuticals

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 Chat Episode 5: NNPDF

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.

Firefly Chat Episode 4: Newborn Screening

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.