Posts

New EU trial regulations could expedite approvals and a...

New EU Clinical Trial Regulations simplify international trials while offering t...

Inborn errors of metabolism elicit unique challenges fo...

As Rare Disease Day is commemorated on 28 February, experts discuss the barriers...

CHMP recommends AbbVie’s Rinvoq be approved for adult GCA

The CHMP has recommended the approval of AbbVie's upadacitinib (Rinvoq) for trea...

Rare disease treatments skipping UK patients largely du...

A survey has shown that pharma companies have low confidence their rare disease ...

Medigene and EpimAb to develop T cell engagers for soli...

Medigene and EpimAb Biotherapeutics have signed a co-development agreement invol...

Why pharma is still far from a treatment for galactosemia

With issues in therapy delivery and a barren trial landscape, short-term chances...

FDA accepts Sobi’s Gamifant application for HLH/MAS tre...

The FDA has accepted Sobi’s sBLA for Gamifant for haemophagocytic lymphohistiocy...

Exelixis focuses on future growth despite COSMIC-313 tr...

At the American Society of Clinical Oncology (ASCO) Genitourinary Cancer Symposi...

Alltrna plans to tackle rare diseases using tRNA-based ...

Alltrna’s CEO Michelle Warner said that the approach allows for basket trials th...

Zevra sells priority review voucher for $150m to suppor...

Zevra will use the $150m to support the commercial launches of rare disease drug...

Rice University Innovators Utilize Gravity to Develop A...

In a groundbreaking achievement, researchers at Rice University’s George R. Brow...

TTUHSC Launches Research Study to Aid Patients Facing T...

The CAVALIER trial, initiated by the Texas Tech University Health Sciences Cente...

Illinois Researchers Unveil Advanced Organic Nanozymes ...

In recent years, the pursuit of environmentally friendly alternatives in science...

St. Jude Researchers Unravel Mechanism Behind Retinoic ...

Neuroblastoma, one of the most common solid tumors in children, poses a daunting...

Next Generation Model: Competition-Driven AI Research S...

The emergence of artificial intelligence (AI) technologies is revolutionizing ho...

Groundbreaking Copper-Infused Microvesicles: Ushering i...

In an extraordinary advancement in the field of dental care, researchers have un...