Dec 17, 2024
Dr. Adrian Quartel, Chief Medical Officer at Zevra Therapeutics, has received FDA approval for their drug MIPLYFFA in combination with miglustat, the first treatment in the US for Niemann-Pick type C, an ultra-orphan disease. This hard to diagnose, rare genetic lysosomal storage disorder affects the recycling of...
Dec 17, 2024
Dr. Adrian Quartel, Chief Medical Officer at Zevra Therapeutics, has received FDA approval for their drug MIPLYFFA in combination with miglustat, the first treatment in the US for Niemann-Pick type C, an ultra-orphan disease. This hard to diagnose, rare genetic lysosomal storage disorder affects the recycling of...
Dec 9, 2024
This is a conversation with Nicole Andrews, a patient with LGSOC and STAAR Ovarian Cancer Foundation Board Chairperson, and Dan Paterson, President and CEO of Verastem Oncology. Low-grade serous ovarian cancer is a rare and slow-growing ovarian cancer that is often misdiagnosed. Verastem Oncology is developing a...
Dec 9, 2024
This is a conversation with Nicole Andrews, a patient with LGSOC and STAAR Ovarian Cancer Foundation Board Chairperson, and Dan Paterson, President and CEO of Verastem Oncology. Low-grade serous ovarian cancer is a rare and slow-growing ovarian cancer that is often misdiagnosed. Verastem Oncology is developing a...
Aug 26, 2024
AJ Tesler, an award-winning producer and director who, along with his wife Jenny, is launching Magnolia’s Guide to Adventuring, a new documentary video series created with support from Acadia Pharmaceuticals, inspired by their daughter Magnolia’s experience with the rare genetic neurodevelopmental disease Rett...