Kristen Kluska

Tazeen Ahmad

Yaron Werber

Yigal Nochomovitz

Anupam Rama

Gena Wang

Jack Allen

Joseph Schwartz

Joon Lee

Liisa Bayko

Luca Issi

Maury Raycroft

Mehdi Goudarzi

Salveen Richter

Christopher Raymond

Dae Gon Ha

Daniel Smith

Ed Arce

Farzin Haque

Hannah Wei

Huidong Wang

Jeff Hung

Laura Chico

Malcolm Kuno

Maurice Raycroft

Maxwell Skor

Michael Riad

Sami Corwin

Shelby Hill

Tommie Reerink

Will Soghikian

Wing Yip

- Ultragenyx Pharmaceutical announced the **first patient has been dosed** in the **_Aurora_ study** (NCT07157254) evaluating **GTX-102 (apazunersen)** for Angelman syndrome.  **[1515673_5ab49631bce3443b95f674fc63789a1a_0]** 
- The _Aurora_ study expands the patient population for GTX-102 to include **younger and older patients** (ages 1 to < 65) and those with **diverse Angelman syndrome genotypes** not included in the fully enrolled Phase 3 _Aspire_ study.  **[1515673_5ab49631bce3443b95f674fc63789a1a_0]** 
- GTX-102 has received **Breakthrough Therapy Designation** from the FDA, and there are **no currently approved therapies** for Angelman syndrome.  **[1515673_5ab49631bce3443b95f674fc63789a1a_1]** **[1515673_5ab49631bce3443b95f674fc63789a1a_2]** 
- Data from the related Phase 3 _Aspire_ study are expected in the **second half of 2026**.  **[1515673_5ab49631bce3443b95f674fc63789a1a_1]**

Ultragenyx Initiates _Aurora_ Study for Angelman Syndrome Treatment

- Ultragenyx Pharmaceutical announced on **October 8, 2025**, that the Italian Medicines Agency (AIFA) has **extended reimbursement for Evkeeza (evinacumab)** for the treatment of Homozygous Familial Hypercholesterolemia (HoFH) in Italy.  **[1515673_dbdf121f56274a2c93034222626d0efb_0]** 
- The extended reimbursement now includes **pediatric patients aged 6 months and older**, expanding from the previous coverage for patients aged 12 years and older, which began in **October 2023**.  **[1515673_dbdf121f56274a2c93034222626d0efb_0]** 
- Evkeeza is a therapy for HoFH, a rare inherited disease causing dangerously high LDL-C, and is administered via **monthly infusion**.  **[1515673_dbdf121f56274a2c93034222626d0efb_0]** **[1515673_dbdf121f56274a2c93034222626d0efb_1]**

Ultragenyx Announces Extended Reimbursement for Evkeeza in Italy

Ultragenyx Pharmaceutical (RARE)