A robust research and innovation ecosystem makes Italy an attractive life sciences hotspot, according to a post from an Italian trade delegation at Bio Digital 2021.
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.
A leader from the genetic solutions firm explains how genetic testing and counseling can help trial teams form more beneficial connections with patients.
An executive from the rare disease technology solutions company discusses how forcing connections with patients can boost research into such conditions.
The company’s cancer drug, devimistat, has received orphan status from the agency for treating clear-cell sarcoma, pancreatic cancer, and other diseases.
Raremark’s patient data and demographics will be integrated in TriNetX’s platform to help people with rare conditions access clinical trials and facilitate recruitment.
Cycle looks to benefit from the advantages of Zydis oral disintegrating tablet technology, developed by Catalent, for four of its drug products treating rare diseases.
The Critical Path Institute and National Organization for Rare Disorders are developing a new data and analytics platform to inform clinical trial design and advance therapies for rare diseases.
Daiichi Sankyo’s Turalio becomes the first FDA-approved TGCT treatment, after receiving breakthrough therapy, priority review and orphan drug designation.
Atlantic Research Group implements Greenphire’s ClinCard and ConneX reimbursement solutions in an aim to ease the clinical trial experience for both sites and patients.
Regulatory actions by the FDA have enabled rare disease drug development to become a compelling space with orphan drug designations and approvals trending upward, says life sciences industry attorney.
More than half of new drug approvals last year were for rare diseases – which face long cycle times and other operational challenges that are necessitating the rapid adoption of new solutions, per a recent Tufts report.
Natural history studies are essential for rare disease research and can potentially replace placebo arms in clinical trials, among several other benefits, says industry expert.
Scientific advances and regulatory incentives are driving the use of genetics in drug development, though the industry’s ability to perform wide-scale testing has outpaced its understanding of the results, the misinterpretation of which can have “disastrous”...
The CRO is adding two new offices in the US to support what has been significant growth over the past several years from pharma and biotech customers working in rare diseases and immune-oncology.
Natural history studies provide researchers with a better understanding of a disease’s progression, supporting clinical trial design, among other benefits, say industry experts commenting on the FDA’s new draft guidance.
WuXi Biologics and CANbridge Pharmaceutical have entered into a long-term partnership to develop and commercialize a portfolio of biotherapeutics for rare diseases.
Centogene and Denali entered a strategic collaboration to identify and recruit patients with a gene mutation to be enrolled in a Parkinson’s disease study.
As aging populations grow and treatment paradigms shift, the industry must prepare for greater challenges in the rare disease clinical trial space, says CRO executive.
The joint drug discovery project announced yesterday will combine Centogene’s genetic testing service offerings with Evotec's iPSC platform and drug discovery capabilities.
Moderna Therapeutics has announced the opening of a state-of-the-art manufacturing site in Norwood, Massachusetts, built to advance their work in the messenger RNA (mRNA) platform, after announcing the plans for the site in 2016.
Ergomed is bolstering its orphan drug development services with its acquisition of PSR Group BV in a deal worth up to €5.7m ($6.69m) – which will position the company to be a “global player” in the area, says CEO.
Researchers and patients have more efficient tools for sharing information on rare diseases, and as knowledge continues to spread, rare disease studies will become less rare.
Eighty-year-old Italian pharma company Recordati believes its
future lies in rare diseases and has put its money where its mouth
is by spending €135m on speciality firm Orphan Europe.
While the procedure for designating drugs for orphan diseases has
improved dramatically in the EU, further progress must be made,
says EU biotech group
