The Batten Disease Support and Research Association welcomed 450 attendees to its annual family conference in Chicago, July 9-12. During the weekend, the BDSRA announced research awards.
The US Food and Drug administration (FDA) has given Orphan Drug Designation to a new dactinomycin formulation in development to treat Ewing’s Sarcoma
Schuller et al evaluated the quality of existing ultra-orphan drug cost-effectiveness and cost-utility data.
Extension data following a Phase 2 study of the drug ISIS-SMNRX continue to show motor benefits for patients with spinal muscular atrophy (SMA).
Each August, the ALS Association plans to host the ice bucket challenge until a cure is found (#EveryAugustUntilACure) for this devastating disease.
A meta-analysis just released in American Heart Journal shows that oral medications for pulmonary arterial hypertension (PAH) seem to reduce symptoms of clinical worsening. As a group, however, they do not appear to extend life expectancy.
The Fast Track program is intended to speed development, review and approval for agents formulated to combat serious and/or life-threatening conditions and unmet medical needs via the facilitation of expanded FDA access and advisory aid.