As news of coronavirus disease (COVID-19) changes hour by hour, one fact remains constant: The IPPF is dedicated to our community during this difficult time, and we are here to provide support and information. We are working hard to keep abreast of the situation and notify you about updates regarding the necessary precautions and recommendations to keep you safe during this pandemic. We will continue to update information on this page as it becomes available.
You may be aware of the IPPF’s advocacy efforts. Now, we need you to advocate, too. Your story is important and shows that rare disease not only affects Americans, but Americans in your district. Your story proves to congressional members that their decisions have a great impact on human life and well-being.
Cabaletta Bio, Inc., a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, announced on January 29, 2020 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company’s lead product candidate, DSG3-CAART, for the treatment of pemphigus vulgaris (PV). DSG3-CAART is designed to target the cause of mucosal PV (mPV), B cells that express pathogenic autoantibodies directed against the DSG3 protein, while preserving normal B cell immune function.
argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, released a strategic outlook for 2020 outlining key priorities for its broad pipeline and path towards achieving its ‘argenx 2021’ integrated commercial vision.
The study met the primary endpoint at week 52 and demonstrated that Rituxan is superior to MMF, with 40.3% of patients treated with Rituxan achieving sustained complete remission (CR) without the use of steroids for 16 consecutive weeks or more, compared to 9.5% in the MMF arm (p<0.0001).
Cabaletta Bio, Inc., a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for the treatment of patients with B cell-mediated autoimmune diseases, announced this week that it has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) to initiate a first-in-human clinical trial of desmoglein 3 chimeric autoantibody receptor T cells (DSG3-CAART) in patients with mucosal pemphigus vulgaris (mPV) to assess the safety and tolerability of DSG3-CAART in these patients.
The IPPF conference was the light and guide that walked us through a very dark tunnel. A tunnel we would’ve walked through alone. But everyone involved turned up the light nice and bright, and we found we were two of many. We were not alone and we would never feel alone again!
Having excellent care and support (and even an infusion center) right here in my neighborhood gives me a sense of security and well-being, and I can trace that support directly back to the IPPF, who helped me find the way. Thank you, IPPF!
Just spending time [at the Patient Education Conference] with people who knew exactly what I was going through, who knew the instantaneous fear and rage that goes along with each new lesion, who were discovering how to find the shady spots at high noon, was an amazing comfort.