News

Oral amoxicillin and intravenous ertapenem kill commensal bacteria and increase risk for antibiotic resistance in Clostridium difficile.

Using realistic diagnostic criteria will increase prevention of tardive dyskinesia.

Today, the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 12-8 in favor of approval of volanesorsen (Waylivra) for the treatment of familial chylomicronemia syndrome (FCS).

On May 17th, the National Organization of Rare Disorders (NORD) will host its 2018 Rare Impact Awards in Washington, DC at the Andrew W. Mellon Auditorium.

TLANDO is being investigated for the treatment of adult men with deficiencies of endogenous testosterone.

The first patient has been dosed in the Phase 1/2 clinical trial of its drug, KB103, for the treatment of dystrophic epidermolysis bullosa (DEB).

Findings from a study uncovered genetic subtypes of DLBCL with distinct genotypic, epigenetic, and clinical characteristics, providing a potential nosology for precision-medicine strategies in diffuse large B-cell lymphomas.

Last night, SELLAS Life Sciences Group, Inc. announced that the U.S. FDA has granted orphan drug designation to galinpepimut-S (GPS) for the treatment of multiple myeloma.

Researchers found that biased language affected a physician’s attitude toward a patient as well as the aggressiveness of their pain management choices.

For even life-altering conditions such as cardiovascular disease, not enough Americans are taking their medicine. Why?

Cellectar Biosciences has announced that the U.S. FDA granted orphan drug designation to CLR 131 for the treatment of rhabdomyosarcoma, a rare pediatric cancer.

Study show blocking FOXM1 can reduce the activation of fibroblasts as well as the process of idiopathic pulmonary fibrosis (IPF) itself.

Astellas Pharma's mirabegron (Mybetriq) will serve as a combination therapy with muscarinic antagonist solifenacin succinate (VESIcare) for the treatment of overactive bladder in patients with particular symptoms.

New data revealed by the Tufts Center for the Study of Drug Development concludes that orphan drug development takes 18% longer than the average time required for other new drugs.

Results show that blade plates or intramedullary nails placed via surgery can be effective treatments for patients with fibrous dysplasia of the proximal femur.

Rare Disease Report sat down with AdAlta CEO Sam Cobb to discuss the impetus for the redesign of AD-214, and what it means for those in the idopathic pulmonary fibrosis.

Biohaven Pharmaceutical has established an expanded access program (EAP) with sublingual BHV-0223 for patients with amyotrophic lateral sclerosis (ALS).

Analyzing post-mortem brain tissue of subjects with major depressive disorder, researchers found that 52 genes were expressed differently in women and men.

Adding an anti-muscarinic to inhaled corticosteroid reduces asthma exacerbations, but possibly not more than other combinations.

The US FDA has granted Palladio Biosciences Investigational New Drug (IND) clearance to proceed with a Phase 2 clinical trial of lixivaptan capsules in patients with autosomal dominant polycystic kidney disease (ADPKD).

DNAtrix will report data from an ongoing Phase 1 trial of DNX-2401 in pediatric patients with newly diagnosed diffuse intrinsic pontine gliomas (DIPG), which indicated efficacy through a prolonged survival rate.

Results from the phase 3 FACETS trial showed that migalastat improved diarrhea in patients with Fabry disease.

Women who consumed fruit just 1-3 times per month versus those that consumed it at least 3 times daily corresponded to a 19% increase in median time to pregnancy.

Alnylam Pharmaceuticals has achieved delivery of novel small interfering RNA (siRNA) conjugates to the central nervous system and intends to advance a pipeline of investigational RNAi therapeutics into clinical development.

The FDA has approved daratumumab (Darzalex) in combination with bortezomib (Velcade), melphalan, and prednisone (VMP) for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT).

Results from the pivotal QuANTUM-R phase 3 study of single agent quizartinib significantly prolongs overall survival compared to chemotherapy in patients with relapsed/refractory AML with FLT3-ITD mutations.

In the recent review, it was shown that surgery can cure nearly all patients with the focal or localized form of congenital hyperinsulinism and prevent complications in patients with the diffuse from in which insulin-producing cells occur throughout the pancreas.

Hemospray, from Wilson-Cook Medical, stopped GI bleeding in nearly all patients within 5 minutes of treatment.

A human clinical trial grant totaling $750,000 was awarded this morning by the Muscular Dystrophy Association (MDA) to Massachusetts General Hospital, with the hope that it will accelerate biomarker research for amyotrophic lateral sclerosis (ALS).

Bruce Wang, MD, a porphyrias specialist and assistant professor in the gastroenterology division in the department of medicine at University of California San Francisco (UCSF) and UCSF Porphyria Center and the Porphyrias Consortium, explains acute hepatic porphyrias (AHPs).