Quiz Results Summary
Firstly, we send our thanks to the CDKL5 caregivers who took the time to respond to our quiz. Although the sample is small, we have identified some insightful take-aways. The vast majority (97%) of CDD caregiver respondents to our quiz said they were interested in accessing gene therapy as a treatment should it become available. This is unsurprising as we know that the burden of a CDKL5 deficiency disorder diagnosis is high and has implications on quality of life for the entire family unit.
We learned that most respondents had a decent understanding of basic drug development in the United States, including what they can do to advance the cause (enrolling in clinical research, participating in registries). However, it should be recognized that the majority did not accurately anticipate the length of time or financial investment it takes to bring a treatment like gene therapy to patients. This is significant because, if expectations are high, but the realities of the drug development timeline and costs are not well understood, disappointment and frustration can set in.
We also learned that most respondents had a good understanding of gene therapy as a technique, but it was very clear that respondents lacked an understanding of the landscape as it applies to treating CDKL5 specifically. The majority of respondents did not know the variety of therapeutic strategies currently being explored for CDD and did not know what challenges are potentially impeding success. Significantly, they were not clear on exactly how many gene therapy projects are currently in the pipeline for CDD.
We know that drug development is complex and, while patients play a crucial role in the development process, they aren't always privy to the details, especially when programs are in pre-clinical development. This is an unfortunate reality and a source of frustration for CDKL5 families who want to be kept in the loop and up-to-date.
- What exactly is being developed for kids with CDD?
- Is anything moving forward?
- Can we get a realistic timeline?
- No questions, just need more transparency.
- Why are so many other disorders getting gene therapy before us?
CDKL5 in Color is working to develop enduring content to educate CDKL5 families. Links in our podcast show notes will point to external resources where families can learn even more. Podcast episode availability on Spotify, Apple Podcasts, and YouTube make the information accessible to a wide variety of families, and short-form video and other social media content increase visibility of what we have to offer.
To families living with a rare disease like CDKL5, no amount of information and insights is too much, and community education should be dynamic and ongoing. Leaders in our CDKL5 community can help families understand the realities of treatment development by having honest conversations about the complexities. CDKL5 in Color is proud to include ourselves amongst the leaders who can support CDKL5 families in this way.
Episode 37 of the CDKL5 in Color podcast (releasing on March 28) tackles the topic of gene therapy. What is known, what is unknown, and how are we meant to make sense of it all? We talk about what we see happening and share our thoughts about these treatments that aim to get at the root cause of CDKL5 deficiency disorder. We also talk about what we can be doing as caregivers to support our loved ones in the here and now while we await the treatments of the future.
Have a listen and let us know what you think!
Until next time 💚
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Disclaimer: CDKL5 in Color (podcast, website, blog, text and visual content, social media, and other creative works) constitutes lived experience and is not intended as medical advice. Always consult your doctor.
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