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Duchenne is a genetic disorder, primarily affecting boys and is known to be fatal. This disease is claimed to affect roughly one in every 3,500 to 5,000 live

Utidelone’s unique properties allow it to cross the blood-brain barrier (BBB), a capability supported by both preclinical and clinical studies. A Phase II study involving utidelone in combination

This sNDA extends the indication of Vemlidy, a once a day 25mg tablet for usage in children aged six years and above weighing a minimum of 25kg with

Sperogenix Therapeutics, the company’s rare disease partner in China, filed the application with the NMPA. The Center for Drug Evaluation (CDE) of the NMPA accepted the filing after

This collaboration is expected to leverage Cipla’s extensive distribution network to enhance the reach of Sanofi India’s CNS products. Under the agreement, Cipla will handle the distribution and

With the deal closing, the merged business will now function under the name Q32 Bio. Concurrent with the merger’s closure, Q32 Bio also finalised a $42m private placement.

This endorsement is a significant step towards the availability of Fabhalta for PNH patients in Europe. The positive opinion is based on data from two Phase III clinical

This development marks the first nonsteroidal medication authorised to treat DMD patients with all genetic variants of a rare neurological disorder. A histone deacetylase (HDAC) inhibitor, Duvyzat can

EVO756 is a highly selective small molecule antagonist targeting mas-related G-protein coupled receptor X2 (MRGPRX2), a receptor implicated in various inflammatory conditions. This partnership builds on a previous