'Beast Games' winner Jeff Allen hopes finding a cure for his son's rare disease will unlock new treatments for other brain conditions

"Beast Games" winner Jeff Allen hopes finding a cure for his son's rare disease will unlock new treatments for other brain ...

First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

Cure Rare Disease and LGMD2L Foundation Announce Multi-Year Partnership to Develop Gene Therapy for Anoctamin 5-Related Disease

Cure Rare Disease (CRD), a non-profit biotechnology organization, today announced a landmark partnership with the LGMD2L Foundation. This collaboration is backed by a generous commitment of $7.65 ...
NC Biotech

Precision BioSciences cleared to begin gene therapy trial for muscular dystrophy

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

Pompe Disease: How This Rare Genetic Disorder Causes Progressive Muscle Weakness and Respiratory Failure

On World Rare Disease Day, we take a look at Pompe disease - a rare genetic disorder caused by a deficiency of the GAA enzyme, leading to glycogen buildup in muscles. It results in progressive muscle ...
Labiotech.eu

Rare Disease Day: is a regulatory overhaul what the therapeutic field needs?

Rare Disease Day is held on the last day of February to raise awareness for rare diseases and address drug development ...
BioSpace

Rare Disease Groups Lobby for Regulatory Expediency as Makary Defends ‘Rigorous’ Approach

This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite ...
University of California

From Rett and Pitt-Hopkins to ALS: 5 rare diseases UC San Diego researchers are tackling in the lab

Researchers at the Sanford Stem Cell Institute have taken the fight against rare diseases to the lab, to patients’ bedsides ...
BioSpace

Cashing In on ‘Creative Fundamentals’ To Fund Rare Disease R&D

Rare disease drug developers struggle to survive in a biopharma investment market that prioritizes large patient populations.
TheHealthSite

Rare Disease Day 2026: Experts Reveal Why Certain Disorders Target Brain And Nervous System

Rare disease day is observed annually on the last day of February to spread awareness on certain conditions that can impact on patients lives.