Another great interview on Brad Loncar’s BiotechTV U - this time with the inspiring Emil Kakkis who successfully developed BioMarin’s Aldurazyme for the rare disorder - MPS I, thus setting the bar for developing treatments for rare genetic diseases. $XBI
#GeneTherapy Biomarin's troubles commercializing hemophilia GT illustrates that patients may prefer...
...managing their disease by titrating non-permanent drugs...
...over taking risk with permanent alteration where the individual does not know where efficacy will exactly land
This week for upcoming IntSculptureDay we are sharing some of the large-scale public artworks that have been created here at NSF. The Giving Tree sculpture, by artist Rachel Doolin, was commissioned to coincide with 10 years of BioMarin in Ireland, and unveiled in April 2022.
A partir de abril… ¡Lanzaremos una edición de la Guía de Terapia Génica en Hemofilia también en inglés! 🙌🏻
🫂 BioMarin Pfizer España CSL SETH Trombosis y Hemostasia FEDHEMO - Hemofilia
BDSRA Database Manager and rare disease sibling Noah Siedman gives updates on last week’s Rare Disease Day at FDA virtual event and his meeting with BioMarin.
Watch the full video: ow.ly/iZwQ50QLa45
#BattenAdvocatesForACure #BattenDisease #RareDisease #Advocacy
