Kevin Freier, a former Pfizer executive, talks about his new podcast ‘Improbable Developments’ that interviews various patients and patient advocates in order to learn more about how they were about to raise awareness about their disease and/or get a medicine developed for their patient populations.
Leticia Orsatti, MD, discusses results from clinical trials evaluating nerandomilast in patients with IPF or PPF.
Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory…
The Priority Review Voucher (PRV) program incentivizes the development of drugs for rare pediatric and tropical diseases.
Tenosynovial giant cell tumor (TGCT) is a rare benign tumor involving the joint synovium, bursae, and tendon sheath.
Matt Winton, PhD, Inozyme Pharma, discusses an analysis characterizing disease severity and progression in patients with ENPP1 deficiency.
Stay informed with our comprehensive calendar of rare disease awareness days, highlighting key dates and resources.
Erin Sullivan, Executive Director of Sisters’ Hope Foundation, discusses her family’s experience with ALSP.
Jolan Walter, PhD, MD, Division Chief of Pediatric Allergy and Immunology at the University of South Florida, discusses genetic testing for WHIM syndrome.
Mike Graglia, Co-Founder & Managing Director of the SynGAP Research Fund, discusses the current landscape of SYNGAP1-related disorders (SRD).
Zoom is the leader in modern enterprise video communications, with an easy, reliable cloud platform for video and audio conferencing, chat, and webinars across mobile,…
