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AATD is a rare genetic disorder that can severely damage the liver and lungs of affected individuals. The trial will start dose escalation in healthy volunteers (Part A)

The randomized, double-blinded, placebo-controlled trial will be conducted at sites in the US, Canada and Europe. The trial is designed to evaluate MM-141 in combination with nab-paclitaxel and

Ora is a US-based global, full-service ophthalmic clinical research and product development company. As part of the deal, Ora will lead the clinical development of PRT2671, and the

The therapeutic potential will be unlocked by combining ArQule’s chemistry and drug development expertise with Beryllium’s discovery platforms, which integrate structure-guided drug discovery, biophysics and cell biology. The

The Irish firm paid $101 per share to acquire Auspex’s portfolio of new medicines for people who live with movement disorders. Auspex’s lead product candidate SD-809 (deutetrabenazine) is

Around 5,000 individuals with systemic lupus erythematosus (lupus) will be enrolled in the trial to help better understand the genetics of lupus. Also involved in this collaboration is

"We are extremely pleased to now have orphan drug designation for MI-100 to treat Stargardt’s disease in place for Europe. This complements and reinforces our development program in

Cortex is a leader in developing drugs for respiratory disorders, particularly sleep apnea (both obstructive and central) and drug-induced respiratory depression. Premature infants exhibit frequent apneic events and

Both studies combine standard doses of gemcitabine or ifosfamide with escalating doses of aldoxorubicin. The combinations appear to be well tolerated, and even at the lowest dose level