Spotlight on Communication: An Interview with Dr. Theresa Bartolotta

Wednesday, April 10, 2024

For Theresa Bartolotta, Ph.D., CCC-SLP, good things have come through community. Bartolotta, a speech-language pathologist (SLP) with more than 40 years of clinical experience, discovered her passion for helping others in high school. She spent many Saturdays volunteering at a respite clinic for children with autism, opening her eyes to career opportunities within the field. … Read More

Advocating for YOU: Rare Disease Week on Capitol Hill

Wednesday, March 20, 2024

IRSF works tirelessly to advocate for you and your loved one with Rett syndrome. Our work takes many forms, our persistence is often unseen, and our successes are the result of countless hours spent meeting with, raising awareness, and educating key decision-makers on the needs of our Rett community. Last month, our advocacy efforts were … Read More

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