Category

Clinics

Neurogene Announces Expansion of Gene Therapy Clinical Trial

Monday, March 4, 2024

Today, Neurogene announced plans to expand and more rapidly enroll patients their Phase 1/2 clinical trial of investigational gene therapy NGN-401. For the full press release, click here. Key Facts: The study will now expand to include a total of 16 pediatric participants (age 4-10). Cohort 1 (low dose group) will increase from 5 to … Read More

Taysha Gene Therapies Adolescent & Adult Study Expanded to U.S.

Thursday, February 29, 2024

Today, Taysha Gene Therapies shared several exciting updates on their REVEAL Phase 1/2 clinical trial of investigational gene therapy TSHA-102: The study will now expand to include adolescent and adult patients in the U.S. (age 12+ protocol). The dosing of the first cohort (low dose) is complete in the adult study in Canada with clinical … Read More

My Rett Ally: Revolutionizing Care Management for Rett Families

Thursday, January 11, 2024

On a recent visit to the emergency room, it took just seconds for Samantha Brant to pull up a detailed, up-to-date medication list for her daughter, Macy. Macy lives with Rett syndrome and Samantha, like all families with a loved one with Rett, must keep meticulous records of health documents to ensure the best possible … Read More

Taysha Gene Therapies Doses First Pediatric Patient in U.S. Gene Therapy Clinical Trial

Wednesday, January 10, 2024

Taysha Gene Therapies today announced the dosing of the first pediatric patient in their REVEAL Phase 1/2 trial of investigational gene therapy TSHA-102. For Taysha’s full press release, click here. Key Facts: The first female pediatric patient was dosed at RUSH University Medical Center in Chicago, an IRSF-designated Center of Excellence. The REVEAL Phase 1/2 … Read More

Neurogene Announces First Pediatric Patients Dosed in US Gene Therapy Clinical Trial

Thursday, November 30, 2023

We’re excited to share that today Neurogene announced that two pediatric patients have been dosed in their Phase 1/2 trial of investigational gene therapy NGN-401. For Neurogene’s full press release, click here. Key Facts: The first two female pediatric patients were dosed sequentially in the 3rd and 4th quarter of this year at Texas Children’s … Read More