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Antibody-Oligonucleotide Conjugate Shows Promise in Myotonic Dystrophy Type 1
Del-desiran reduced DMPK mRNA levels, but led to two serious adverse events ...
Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule ...
VOXZOGO® (vosoritide for injection) is the only medicine indicated to increase linear growth in children with achondroplasia ...
Two new drug applications (NDA) for ulixacaltamide in essential tremor (ET) and for relutrigine in SCN2A and SCN8A developmental and epileptic ...
Discusses Positive Phase 3 PROPEL 3 Results for Infigratinib in Children With Achondroplasia February 12, 2026 8:00 ...
What began as a search for hormone-processing enzymes ultimately transformed the treatment of high cholesterol.
Two new drug applications (NDA) for ulixacaltamide in essential tremor (ET) and for relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) have been submitted to the U.S.
Two UT Southwestern Medical Center researchers have identified a protein that plays a key role in controlling the liver's release of cholesterol-carrying lipoproteins into the bloodstream, a discovery ...
In the modern world, the human body is besieged by a silent category of chemicals that interfere with one of its most ...
Solid Biosciences Inc. is a Strong Buy driven by positive FDA alignment on phase 3 IMPACT DUCHENNE trial for SGT-003. Read ...
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