Pociredir 12 mg dose has shown favorable safety and signs of efficacy in a Phase 1b trial of sickle cell disease (SCD).

Medications that promote repair of the myelin sheath that protects nerve cells may help limit damage due to MS, a trial ...

In a small trial across ten U.S. research sites, a novel HIV vaccine candidate has shown a result that has eluded scientists ...

Women in their childbearing years are significantly less likely than men in the same age range to be given disease-modifying ...

The FDA has granted breakthrough therapy designation to DYNE-251 for the treatment of DMD in patients amenable to exon 51 ...