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  • PHILADELPHIA – iECURE, a mutation-agnostic in vivo gene editing company striving to cure devastating diseases with high unmet need, today announced it has appointed Brian Di Donato, MBA to its Board of Directors. Mr. Di Donato has more than 20 years of financial and leadership experience and currently serves as...
  • BOSTON and LONDON – Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the appointment of two gene therapy and industry experts, Fulvio Mavilio, Ph.D., and Nicoletta Loggia, Ph.D., to serve as chief scientific officer and chief technical officer, respectively. The company also announced the promotion of Leslie Meltzer, Ph.D.,...
  • BAAR, Switzerland and MOUNTAIN VIEW, Calif. – atHeart Medical, a medical device company dedicated to establishing the new standard of care for closure of atrial septal defects (ASD), today announced the appointment of Andrew Cleeland to the company’s Board of Directors. With over thirty years of experience, including specific expertise bringing game-changing technologies to market, Mr....


Rare disease spotlight


Are you an advocate for people living with a rare disease? Take our free online training and learn how to:

☑️ Create empowered communities
☑️ Build effective channels and reach the right people.

Learn more:

Help raise awareness of and support #RareDisease at the virtual #RAREsummit21 brought to you by @Camraredisease on 7 October 2021. Agenda, tickets and more info at

Thank you Senator @JimInhofe for cosponsoring the #ACTforALS (S. 1813), which would speed up therapies for the #NMD community, including those living with #ALS! #EndALS

This Fact Friday we are highlighting studies on DMD affected females! Check out these interesting quotes from 2 research articles on DMD carriers. #Duchenne #MuscularDystrophy #RareDisease #NotJustCarriers #DMD

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