Navigating Clinical Trials: A Guide for Rare Disease Patients

Navigating Clinical Trials: A Guide for Rare Disease Patients

A condition is defined as a rare disease if it affects fewer than 200,000 individuals. Approximately 7,000 conditions meet this definition. In 2016, the United States Food and Drug Administration awarded $23 million dollars towards a four year period that supported research for 21 different rare diseases. However, most rare diseases lack disease-modifying therapies or effective medications for several reasons…

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AI in Healthcare: Revolutionizing Genomic Sequencing and Rare Disease Treatment

AI in Healthcare: Revolutionizing Genomic Sequencing and Rare Disease Treatment

In recent years, artificial intelligence (AI) has made significant strides across various industries, but its impact on healthcare, particularly in the realm of genomic sequencing and rare disease research, is transformative. As we continue to navigate the complexities of the thousands of rare diseases affecting millions worldwide, AI offers unprecedented opportunities to revolutionize diagnosis, treatment, and patient care.

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2024 Informa Connect Rare Disease Innovation and Partnering Summit

The 2024 Informa Connect Rare Disease Innovation and Partnering Summit is held this year between March 19th-21st. The summit will be a space that brings together organizations focused on rare diseases and orphan drug development with patient advocates, patient support/services, market access, and technological development. The summit is an opportunity to be exposed to new information and resources, discuss important issues and their solutions, and hear from patient’s voices that are driving drug discovery in the rare disease realm. 

If you're also looking to connect with industry representatives, nonprofits, patients, and vendors, the summit will be a chance to help improve the quality of life for members in the rare disease community through education and advocacy. Held in Philadelphia, the summit will also host relevant experts and multidisciplinary stakeholders, including the FDA, Rare Revolution, Cornerstone Pharmaceuticals, and more. Key topics will be covered, including "Rapid Fire Case Studies Directly from the Thought Leaders Innovating in the Rare Disease Space" presented by Rare Genomics co- founder and Sudo BioSciences CBO Dr. Imran Barbar, and "Combatting Mental Health Challenges within Rare Disease," featuring founder and CEO of Angel Aid, Cristol Barrett O’Loughlin.

Register for the conference here: https://informaconnect.com/rare-disease-summit/pricing/

RG Rare Disease Day Acknowledgment: Small steps make a big difference

RG Rare Disease Day Acknowledgment: Small steps make a big difference

LOS ANGELES, CA; April 15, 2019 --

Thank you to all our supporters on **Rare Disease Day 2019**, we are so thankful to have a community that is working with us to push science and medicine forward to meet the needs of rare disease patients.

This year we were lucky to have a new community raise awareness with us on Rare Disease Day. We want to thank all Illumina employees who decided to advocate for rare disease patients on this important day. When we say that every bit makes a difference, that is because it makes all the difference, especially for families with significant unmet need.

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