Expanding Access to Genome Sequencing in Rural Populations & More — This Week in RARE Daily
April 18, 2024
This Week in RARE Daily is a feature from Global Genes where you can get a quick rundown of the top 5 headlines in the rare disease space from our editorial staff. Here are top stories from this past week, April 12-18, 2024:
Expanding Access to Genome Sequencing in Rural Populations
Patients in rural area often face challenges accesses cutting-edge medical technologies, such as state-of-the-art genome sequencing, but a new pilot study from the Children’s Mercy Research Institute offers a framework for how to improve access and care for patients far from metropolitan areas.
A 37-Year Path to a Diagnose, but Now a Drug May Be Two Weeks Away
Courtney Ampezzan waited 37 years to get a diagnosis for WHIM syndrome, but the FDA is expected to decide whether to approve X4 Pharmaceuticals’ mavorixafor by the end of April.
Group Calls for Use of Genome Sequencing as First Line Test for Patients Suspected of Genetic Disease
The Medical Genome Initiative, a group made up of sequencing giant Illumina and leading clinical genomics laboratories, has called for the use of genome sequencing as a first line test for patients suspected of having a genetic disease.
Two Rare Disease Drug Developers Raise Combined $152.5 Million in PIPE Offerings
Two companies developing therapeutics for rare diseases, Zura Bio and Benitec Biopharma, raised $152.5 million in capital. The financings reflect an ongoing trend among biotech investors to capitalize on buying private placements in public companies to capitalize on depressed values in public markets.
Across Healthcare Enters Collaboration with n-Lorem Foundation to Develop Submission Platform for Docs
The nonprofit foundation n-Lorem has entered into a collaboration with a technology company to develop a first-of-its-kind submission platform and system that allows research physicians from across the United States to submit applications for treatment for their patients with rare illnesses with 30 or fewer known patients.
Stay Connected
Sign up for updates straight to your inbox.