Ambros Therapeutics launches with $125 million to advance breakthrough therapy for rare pain disorder

The funding will support the pivotal Phase 3 clinical trial of neridronate in CRPS-1

Praxis Precision hits milestone in fight against rare paediatric seizures

The EMBOLD study evaluated relutrigine for patients with SCN2A and SCN8A developmental and epileptic encephalopathies

Bayer advances fight against rare kidney disease with new experimental drug

Alport Syndrome is caused by genetic mutations affecting type IV collagen in the kidneys, ears, and eyes

Belite Bio reports landmark phase 3 success for Stargardt Disease therapy

Seeing well-controlled Phase 3 data that shows a marked slowing of lesion growth in Stargardt disease is deeply encouraging

Merck KGaA’s Cladribine capsules gain FDA fast track for rare neuromuscular disorder

FDA grants accelerated approval to sevabertinib for non-squamous non-small cell lung cancer

HER2-mutant NSCLC typically has a poor prognosis and limited treatment options

Cardiol Therapeutics wins US patent protection for heart-disease drug platform

The patent will lock in broad protection for the company’s lead therapy CardiolRx and its novel subcutaneous candidate CRD-38

Zydus receives FDA’s ODD for Desidustat for the treatment of beta-thalassemia

Orphan drug designation (ODD) by the USFDA for Desidustat, provides eligibility for a potential seven-year marketing exclusivity subject to the USFDA approval

FDA approves UCB’s Kygevvi for rare genetic mitochondrial disease TK2 deficiency

Kygevvi combines two pyrimidine nucleosides, doxecitine and doxribtimine, which act by integrating deoxycytidine and deoxythymidine into skeletal muscle mitochondrial DNA

Sanofi’s Efdoralprin Alfa shows superiority in phase 2 Alpha-1 antitrypsin deficiency study

Efdoralprin Alfa was well tolerated, with a safety profile comparable to plasma-derived therapy