In a major partnership aimed at accelerating research into Rett syndrome, a rare neurological disorder predominantly affecting girls, digital healthcare solutions company Vivalink, has joined forces with the Rett Syndrome Research Trust (RSRT).
In a seminal move coinciding with Rare Disease Day, Emmes Endpoints Solutions has taken a significant step forward in the realm of rare disease research.
Biogen Inc. has announced a significant advancement in the treatment of amyotrophic lateral sclerosis (ALS), commonly known as motor neuron disease (MND).
Biogen Inc. has taken a step closer to bringing its ‘groundbreaking treatment’ to patients with a rare, genetic, life-shortening, debilitating, and neurodegenerative disorder.
Inceptua Group and Cycle Pharmaceuticals partnership will give access to Free Goods Partnership that gets medicine to patients with extremely rare genetic conditions, which they would be unable to access otherwise.
OSP was delighted to speak to Galina Nesterova, executive medical director at the Rare Disease & Pediatrics Center of Excellence, and Susan McCune, vice president of pediatrics and clinical pharmacology both within medical science and strategy at...
The first and only enzyme replacement therapy for the treatment of a very rare and complex blood disorder has been approved by the US Food and Drug Administration.
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).
The treatment of rare brain disease, progressive supranuclear palsy (PSP), that has no disease modifying therapies, has been given a glimmer of hope by Amylyx Pharmaceuticals.
Former UK Prime Minister, David Cameron, will lead the international efforts of The Oxford-Harrington Rare Disease Centre's new initiative, the therapeutics accelerator.
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...
Progressive supranuclear-palsy (PSP) is a very rare brain disease and until recently, it was almost unheard of. Along with many neurodegenerative diseases, it has a high unmet medical need.
A licensing agreement has been signed that will give Spanish pharma company, Ferrer, worldwide rights to a treatment for progressive supranuclear palsy (PSP).
Father and software engineer, Vincent Keunen combined his personal experience of leukemia and his son's bone cancer with his professional skills to provide all patients with a tool to manage their health information on their smartphone.
Cardiff-based CatSci is investing in its oligonucleotides capability that will enable the company to make oligos from nanomole quantities up to 30 grams using solid phase synthesis.
Outsourcing-Pharma caught up with Esther Mahillo, VP of Operational Strategy and Feasibility, Precision for Medicine to talk about advancements in patient enrollment in oncology and rare disease clinical trials.
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.
The biotechnology company has reported its candidate for treating amyotrophic lateral sclerosis patients has shown promising results in an early-phase study.
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 Australian biotechnology company has been granted ODD status from the agency for Veyonda, a therapy intended for the treatment of soft-tissue sarcoma.
The company (which specializes in developing therapies for rare cancers) currently is working on a number of candidates, including its lead drug CPI-613.
The recent Outsourcing-Pharma webinar hosted a trio of industry experts highlighting challenges, opportunities, and innovations in the rare disease realm.
With a specific focus on clinical trials involving treatments for rare diseases, Orphan Reach seeks to build upon the services that conventional CROs offer.
Ergomed, a global contract research organization, is partnering with Trialbee to improve patient recruitment at its new Rare Disease Innovation Center.
The AI-centered genomics company is joining with Don’t Forget Morgan, a group for patients with BPAN, to explore treatments for the neurodegenerative condition.
The pharmaceutical company’s Lilly Institute for Genetic Medicine reportedly will focus on developing RNA-based therapeutics in a ‘state-of-the-art’ facility.
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 Stoneygate and Kidney Research UK Alport Research Hub aims to accelerate research and develop treatments for Alport syndrome, a rare genetic disorder.
Professionals from various corners of the life-sciences field share views on significant challenges of 2021, bright spots, and what to look for in 2022.
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.
The two firms will work together on solutions to connect de-identified, first-party data to real-world data to help advance precision medicine development.
During the Rare/Orphan Diseases, Special Patient Population webinar, a group of industry experts discussed challenges and opportunities faced in the field.
The organization, focused on building awareness and finding therapies for the rare disease, is boosting its annual research funding to more than $2.7m USD.