An Update From Ultragenyx On Their UX055 Gene Therapy Program for CDD
In November of last year, we shared a letter from Ultragenyx about their UX055 Gene Therapy Program for CDKL5 Deficiency Disorder in which it was stated, “Pending the outcome of additional non-clinical studies, we expect to submit an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) in 2023.”
Today, we would like to share an update on the timing of the IND filing. This update notifies the community of a delay in the filing that will push it beyond Q4 of 2023. Please note that Ultragenyx is urgently working towards a future regulatory submission and will provide an update by March 2024 when new information becomes available.
Did you know that #GivingTuesday is our single largest fundraising day of the year? That's not because of us, it's because of YOU!
Special this year, we have a fundraising match of $25,000 thanks to a generous anonymous donor! Join our Facebook fundraising challenge and help us match $25,000 from now until the end of the year!
Share your CDKL5 story and let the people in your network know why their support for the IFCR makes a difference for CDKL5!
Not on Facebook? It's ok! Donations that come through our website count for the match too! Let's work together to reach our collective goal of $25,000!
November is Epilepsy Awareness Month We have resources for spreading awareness about epilepsy and CDKL5 available for download on our website. Find profile photo frames and facts to share!
November is Family Caregivers Month "During National Family Caregivers Month, we recognize the love and sacrifice of more than 50 million Americans providing crucial care and medical assistance to parents, children, siblings and other loved ones, ensuring their health and dignity... Their work is a profound service to their families and to our nation, but they are still too often unseen, undervalued and unpaid." - US Census Bureau
Caregiving for someone with CDD is a full-time job. We have resources to support caregivers such as family support and resource guides for how to live the best life possible with this diagnosis. Find it all on our website.
Ztalmy Webinar
November 8 | 7 pm ET - Join Marinus for an educational webinar to listen, learn, and ask questions about Ztalmy - the first and only treatment indicated specifically for seizures associated with CDD in people 2 years of age and older.
This virtual program will include education from a healthcare provider, as well as real experiences related to diagnosis and treatment from a caregiver of a loved one living with CDD and prescribed Ztalmy. Click to register.
Family Support Program
We know how hard life with CDKL5 can be and we are here for you whenever you need us.
Would you like to speak with a fellow CDKL5 parent who knows what you're facing? We have peer support volunteers who are available to offer a listening ear or resources that can help. Email FSR@CDKL5.com.
Did you know? Our website is a great place to learn what clinical trials are enrolling for CDKL5!
There are opportunities for interventional trials as well as clinical research, including ways to participate in CDKL5 research from your home, anywhere in the world!
This information and even more can be found on our website. Check back frequently for updates as new opportunities emerge!
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Have you joined "Connect CDKL5"? Connect families receive direct communication about clinical trial opportunities for CDKL5 and other important information important for families. Join today!
