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Let s Get to Know Paula Ragan, CEO and President of X4 Pharmaceuticals

Let s Get to Know Paula Ragan, CEO and President of X4 Pharmaceuticals
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Pivots During Pandemic Bring Patient-Centric Options to Rare Disease Patients

Published: Mar 01, 2021 By Heather McKenzie February 28 marked Rare Disease Day, and biotech companies gathered together to raise awareness, advocate for improved access to treatment and let patients know how they are pivoting to improve patient care and access during the COVID-19 pandemic and beyond. Rezolute Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for patients with metabolic and orphan diseases, was just beginning a Phase IIb study of its lead candidate, RZ358, for Congenital hyperinsulinism (HI), when the chaos struck. “We have hospitals around the world where the COVID ward was right next to the research ward. We had hospitals that for six months shut down entirely,” shared Erin O’Boyle, Head of Clinical Operations at Rezolute. “We had a lot of overnight stays, but then, right when we were kicking off the study and going into enrollment and starting to activate our sites, COVID hit, and it really kind of changed the

Michele rheePaula raganTay sachs sandhoffErin oboyleGavin corcoranPatient affairsRezolute incRare diseaseRare disease dayErino boyleClinical operationsGene therapiesSio chief rd officerChief executive officerVice presidentPivots during pandemic bring patient centric options to rare disease patients

X4 Pharma Adjusts Clinical Trials For a COVID World

X4 Pharma Adjusts Clinical Trials For A COVID World By Ed Miseta, Chief Editor, Clinical Leader Follow Me On Twitter @EdClinical X4 Pharma is a company focused on delivering innovative treatments for patients with rare diseases of the immune system. With the COVID pandemic afflicting patients around the world, those with a compromised immune system were at even greater risk. The company is currently conducting a global Phase 3 trial that was underway when the pandemic hit. The trial is for patients with WHIM Syndrome, who are born immunocompromised. The study is particularly demanding as it is being run in 17 different countries.

Paula raganEd misetaChief editorபால ராகன்தலைமை ஆசிரியர்

X4 Pharmaceuticals Appoints Diego Cadavid, M D , as Chief Medical Officer

Share: BOSTON, Dec. 14, 2020 (GLOBE NEWSWIRE) X4 Pharmaceuticals, Inc. (NASDAQ:XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today announced the appointment of Diego Cadavid, M.D., as Chief Medical Officer. Dr. Cadavid will be responsible for the strategy, direction and execution of the company s clinical development programs. We are very pleased to welcome Diego to our team, said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. We believe his strong clinical development expertise, in particular with the advancement of small molecules and biologics for the treatment of rare and immunological diseases, will be invaluable as we continue to advance our ongoing clinical programs, including our Phase 3 trial of mavorixafor in WHIM syndrome and our Phase 1b studies in Waldenström s macroglobulinemia and severe congenital neutropenia, as well as our preclinic

United statesSan antonioUnited kingdomUniversity of texasPaula raganPontificia universidad javerianaDiego cadavidMonica rouco molinaArmed forces institute of pathologyHead of clinical development at fulcrum therapeuticsUniversity of texas health science centerExchange commissionLifesci communicationsGeorgetown universityPharmaceuticals incChief medical

X4 Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for Mavorixafor for the Treatment of WHIM Syndrome

BOSTON, Dec. 10, 2020 (GLOBE NEWSWIRE) X4 Pharmaceuticals, Inc. (NASDAQ:XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today

United statesPaula raganRouco molinaExchange commissionLifesci communicationsEuropean commissionDrug administrationPharmaceuticals incRare pediatric diseaseChief executive officerFast trackFast track designationBreakthrough therapy designationOrphan drugPrivate securities litigation reform actஒன்றுபட்டது மாநிலங்களில்