Schultz, Anastasia
Cheng, Shun-Yun
Kirchner, Emily
Costello, Stephanann
Miettinen, Heini
Chaverra, Marta
King, Colin
George, Lynn
Zhao, Xin
Narasimhan, Jana
Weetall, Marla
Slaugenhaupt, Susan
Morini, Elisabetta
Punzo, Claudio
Lefcort, Frances
Funding for this research was provided by:
Familial Dysautonomia Foundation
National Institutes of Health (NEI R21EY031130, R01EY029544)
The Sloan Indigenous Graduate Partnership Program
Montana INBRE Native American Graduate Fellowship
National Institutes of Health, Diversity Supplement (3R01DK117473-02S1)
PTC Therapeutics
Article History
Received: 20 May 2023
Accepted: 18 October 2023
First Online: 30 October 2023
Competing interests
: The authors declare competing financial interests. Xin Zhao, Jana Narasimhan, and Marla Weetall are employees of PTC Therapeutics, Inc., a biotechnology company. In connection with such employment, the authors receive salary, benefits, and stock-based compensation, including stock options, restricted stock, other stock-related grants, and the right to purchase discounted stock through PTC's employee stock purchase plan. Susan A. Slaugenhaupt is a paid consultant to PTC Therapeutics and is an inventor on several U.S. and foreign patents and patent applications assigned to the Massachusetts General Hospital, including U.S Patents 8,729,025 and 9,265,766, both entitled “Methods for altering mRNA splicing and treating familial dysautonomia by administering benzyladenine,” filed on August 31, 2012 and May 19, 2014 and related to use of kinetin; and U.S. Patent 10,675,475 entitled, “Compounds for improving mRNA splicing” filed on July 14, 2017 and related to use of BPN-15477. Elisabetta Morini and Susan A. Slaugenhaupt are inventors on an International Patent Application Number PCT/US2021/012103, assigned to Massachusetts General Hospital and PTC Therapeutics entitled "RNA Splicing Modulation" related to use of BPN-15477 in modulating splicing. No other authors declare any competing interests.