Neurogene Workshop Seeks Families Affected by CLN5

Neurogene Workshop Seeks Families Affected by CLN5
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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.

Those interested in joining a workshop should contact Gay Grossman at [email protected] or 858–472–6947.

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.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
Total Posts: 14
Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.
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Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
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