Editor’s note: This story was updated March 30, 2021 to clarify that Neurogene is hosting videoconferences workshops for families in any country.
Neurogene, which is developing treatments for Batten disease and other rare neurological disorders, is inviting families affected by CLN5, a form of late infantile Batten, to participate in a virtual caregiver focus group. The meeting provides a forum for families to share their experiences and perspectives, and learn from one another.
The biopharmaceutical company is hosting multiple Zoom videoconferencing workshops and engaging with families from any country, taking into account interested families’ availability to accommodate various time zones, and already has presented at least one. Participants will be paid $375.
For Neurogene, the overarching goals are to learn from parents and caregivers about their child with CLN5, particularly about the disease’s motor and vision aspects. The company wants to know how these symptoms manifest in participants’ children, and how they affect the family and patients’ day-to-day lives.
Late infantile Batten disease typically affects children ages 2 to 4 and is characterized by seizures and loss of motor skills and cognitive ability. Children with CLN5 often have normal development until the first symptoms, which usually are problems with movement and developmental regression. Vision loss and speech problems are other disease features.
Information gleaned from the workshops will be used internally for learning purposes and will not be shared publicly. It also should help inform the company’s future CLN5 gene therapy clinical development plan. The U.S. Food and Drug Administration last July designated the therapy, which seeks to deliver a working copy of the human CLN5 gene, an orphan drug to potentially treat the disorder. The status is intended, through special incentives, to encourage the development of treatments for rare diseases.
“If your family is presently impacted by CLN5 or has been previously, we encourage you to consider participating,” the company stated in its invitation.
During the roughly two-hour workshop, participants have the opportunity to learn about the company, engage openly about their family’s journey, and share their thoughts and expectations about gene therapy trials being developed and Neurogene’s support and education.
Before each workshop, Neurogene will send participants materials to read, including questions they will be asked. Notes will be taken on the calls, which will not be recorded.
Workshop participation will have no bearing on whether participants’ children are enrolled in any future Neurogene gene therapy clinical trials.
Batten disease is thought to affect two to four of every 100,000 U.S. residents, and roughly one in every 100,000 individuals globally.
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