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  • PASADENA, Calif. – Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced ARO-DUX4 as Arrowhead’s first muscle targeted investigational RNAi therapeutic candidate to utilize its proprietary Targeted RNAi Molecule (TRIMTM) platform. ARO-DUX4 is designed to target the gene that encodes human double homeobox 4 (DUX4) protein as a potential treatment for patients...
  • SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today introduced a new no-charge, patient-initiated testing program in the United States for individuals who have symptoms consistent with, or a diagnosis of, cerebral palsy (CP) with no evidence of brain injury. CP is a group of disorders that affect a person’s ability to...
  • Curing cancer has been the dream of countless doctors and researchers over the decades. Yet some types of cancer, whether because they affect relatively few people or lack influential advocates, receive less attention and funding than others. A recent gift to Harvard Medical School (HMS) is boosting efforts to understand...

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#OneRbWorld2021 Virtual has a NEW DATE: 1-3 October. We’re sad COVID keeps us apart again, but we promise you a very warm welcome and fully interactive program. Please save the NEW DATE, 1-3 Oct for immersive #retinoblastoma community and collaboration to advance care for all.

Proud to partner with #PatientAdvocacy organization @CureCRB1 in our 2021 Young Investigator Draft to co-fund important research for the #RareDisease community! #WeTackleRare

New #webinar: "Transitioning to Adult Care” on April 21 at 2:00pm ET. Join #NORD to learn how to plan for the transition from parent-supervised pediatric care to more independent adult care. #rarediseases Register: http://bit.ly/3dm5YXT

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