Unfortunately, more than 700 rare diseases affect more than 30 million people in the US alone and with many of them being life threatening, it is still a concern that most still do not have treatments.
OSP had a quickfire interview with Rebecca Starkie, senior global patient engagement director, at Advanced Clinical where she explains the challenges faced by rare disease patients - and suggests ways to improve.
Element Biosciences, Inc., will share news of its sequencing methodology and host a workshop on higher accuracy sequencing for improved variant detection in Washington DC from today.
A transaction between Citius Pharmaceuticals and TenX Keane which will result in the formation of Citius Oncology, Inc. is due to close in the first half of next year (2024).
Acasti Pharma, a late-stage biopharma company with drug candidates addressing rare and orphan diseases, has selected WuXi Clinical Development to conduct its STRIVE-ON phase 3 safety trial for GTX-104, a novel injectable nimodipine formulation for intravenous...
A licensing agreement has been signed that will give Spanish pharma company, Ferrer, worldwide rights to a treatment for progressive supranuclear palsy (PSP).
During the World Orphan Drug Congress, a co-founder of the CRO outlined how patient perspective can be vital to better results in rare-disease research.
Epistemic AI, Cincinnati Children’s Hospital Medical Center, and Boomer Esiason Foundation are working together to advance research into the rare disease.
The CRO and medical genetics company will collaborate to accelerate trials centered on rare diseases, using real-world data and genetic testing technology.
The recent Outsourcing-Pharma webinar hosted a trio of industry experts highlighting challenges, opportunities, and innovations in the rare disease realm.
In its latest diversity, equity and inclusion report, Global Genes offers insight into obstacles faced by rare-disease patients from minority communities.
On February 23, five days ahead of the day of rare-disease awareness, OSP’s Rare and Orphan Diseases webinar will share insights from top industry experts.
During OSP’s exclusive February 23 webinar Rare and Orphan Diseases, leading expertswill lend their unique experience and expertise to the important topic.
The advocacy group is joining with the Rare Disease Diversity Coalition on an initiative to identify and overcome challenges faced by minority patients.
Two experts from the contract research organization offer advice on planning and executing clinical studies with the unique needs of such patients in mind.
Stakeholders in the Rare Disease Cures Accelerator-Data and Analytics Platform initiative are partnering in hopes of bringing treatments to patients faster.
The advocacy organization's PIE4CNS initiative is aimed at targeting and reducing gaps in diagnosis, research, and trials for rare neurological conditions.
The CRO has launched an initiative that offers help navigating regulatory issues for ultra-rare disease patient advocacy groups that demonstrate a need.
During the Rare/Orphan Diseases, Special Patient Population webinar, a group of industry experts discussed challenges and opportunities faced in the field.
A leader from the global CRO discusses how putting the patient first in clinical research is essential to discovering new therapies for orphan diseases.
A father of two boys with Duchenne muscular dystrophy is running the Austin Marathon to raise funds toward pursuit of a cure for the rare, fatal disease.
The free Patient-Centric Trial Development Toolkit is aimed toward helping sites and sponsors identify and minimize risks in rare-disease clinical studies.
A representative of the rare-disease specialist firm outlines the unique obstacles of orphan CNS disease research, and how professionals can overcome them.
The IQVIA and NORD study shows rare-disease treatments account for only 11% of new drug spending, but 80% of orphan products treat rare diseases alone.
