Zevra to receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposiumEight abstracts discussing data and clinical ...

Patient advocacy groups have written to Union Health Minister J.P. Nadda, raising concerns about the delay in rolling out the ...

Chiesi GRD has an initial focus in developing therapies for lysosomal storage disorders (LSDs), including Fabry disease, as well as other rare diseases in haematology and opthalmology indications.

JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR") announced today that it will present at the 21st Annual WORLDSymposiumtm 2025, held February 3-7, 2025, in San Diego, Calif. The poster presentations ...

Patient advocacy groups warned that delays in implementing India's rare disease policy have left many, including children, in ...

Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will feature oral and poster presentations at the upcoming ...

Visualizing cells after editing specific genes can help scientists learn new details about the function of those genes. But ...

Jan. 22, 2025 — Following the lead of previous work on mRNA-based vaccines, therapies for spinal cord injuries and more, the method relies upon mineral-coated microparticles to deliver mRNA that ...

Denali Therapeutics (NASDAQ:DNLI – Free Report) had its target price trimmed by The Goldman Sachs Group from $45.00 to $40.00 in a research report sent to investors on Tuesday morning,Benzinga reports ...

But this data can be saved if you have secure backups. One such way to create reliable backups is by using cloud storage that doesn't rely on external devices. We’ve rounded up the best cloud ...

June 10, 2024 — Scientists have grown 'mini-guts' in the lab to help understand Crohn's disease, showing that 'switches' that modify DNA in gut cells play an ... Major Cause of Inflammatory ...

Julien Grogg, PhD, MBA - CEO of Release Therapeutics Release Tx logo Dr. Julien Grogg will Lead the Company's Transition to Clinical ...