Clinical Trials Arena on MSN
Ultragenyx’s gene therapy shows sustained improvements in MPS IIIA
There are currently no approved disease-modifying therapies for MPS IIIA.
A missing brain molecule may be disrupting neural wiring in Down syndrome, according to new research. Replacing it in adult mice rewired brain circuits and improved brain flexibility, challenging the ...
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that the first participant has been enrolled in the BLOOM Phase 2 ...
After its cannabidiol gel candidate failed a phase 3 trial in fragile X syndrome, Harmony Biosciences has now paused development of the asset in a different genetic disease. Harmony announced in ...
X is launching a marketplace for inactive handles that will allow Premium subscribers to request and purchase usernames. The marketplace will offer both free and paid options for requesting or ...
New research suggests a missing brain molecule may hold the key to understanding – and potentially treating – the faulty neural circuits seen in Down syndrome. Restoring the molecule, called ...
Harmony Biosciences Provides Update From Its Phase 3 RECONNECT Study of ZYN002 in Fragile X Syndrome
PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)--Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced topline results from its Phase 3 registrational clinical trial (the RECONNECT Study) of ...
Servier is souping up its neurology pipeline with a clinical-stage candidate sourced from U.K. biotech Kaerus Bioscience for a rare genetic disease. The French pharma is offering as much as $450 ...
Les Laboratoires Servier said that it agreed to acquire a potential treatment for Fragile X syndrome (FXS), the most common genetic cause of autism spectrum disorder (ASD), from its original developer ...
Researchers at The Hospital for Sick Children (SickKids) have uncovered a promising new therapeutic target for Barth syndrome, a rare genetic condition with no current cure. Barth syndrome is an ...
Researchers led by UC Davis Health scientist Sanchita Bhatnagar have developed a promising gene therapy that could treat Rett syndrome. The therapy works on reactivating healthy but silent genes ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results