22nd Orphan Drugs and Rare Diseases Global Congress 2024 Americas
Enhance Information Sharing, Improve Collaboration to Increase Drug Approval for Rare Diseases
Boston, MA, USA
Thursday 14th - Friday 15th November 2024
Bionical Emas
Bionical Emas is the only Contract Research Organization (CRO) to combine Clinical Development, Clinical Trial Supply (CTS) and Early Access Programs (EAP) bringing life-changing medicines to patients around the world. Their unique integrated business model, with its range of services and capabilities, benefit many of the world’s leading pharma and biotech companies. This distinctive offering enables them to maximize access and generate evidence at every stage of the drug development pathway. Bionical Emas has extensive experience across all phases of clinical research, with a history in rare disease, oncology/heme-oncology and infectious diseases. The company has an established global footprint across the US, EU and Australasia markets and has over 200 employees based around the globe, supporting many thousands of patients. The Bionical Emas vision is to maximize access and generate evidence across the entire clinical development pathway, positively impacting current and future patients.
Pulse Infoframe
Pulse Infoframe is a pioneer and leading provider in the life science industry with a real-world evidence (“RWE”) generation platform enabling acceleration of scientific research and the development of new therapeutic products. Pulse is impact-focused on ensuring that fit for purpose critical knowledge and insights enable every phase of the drug development process for (i) oncology including skin cancers, Melanoma, Lung, and Ocular Melanoma and Castleman Disease; (ii) ultra-rare and rare diseases such as Lysosomal Storage Diseases, CDKL5-deficiency Disorder, ( iii) PBC and pruritis, (iv) Alport Syndrome, and (v) Narcolepsy. Pulse’s enterprise-grade platform adaptive technology is trusted by a global, ‘blue-chip’ customer base of top pharma companies, patient advocacy groups and academic medical centres.
PTC Therapeutics
PTC is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. Link: www.ptcbio.com