News

The injection therapy was a previously non-approved FDA product that was grandfathered into marketed use for about 4 decades.

Soleno announced this morning that its multi-center Phase 3 clinical trial of Diazoxide Choline Controlled-Release (DCCR) tablets for the treatment of Prader-Willi Syndrome has been initiated. The potential therapy is first being evaluated at the Seattle Children’s Hospital.

The rate shows a correlated effect on the collaboration efforts between nurses and physicians.

The prevalence of disordered eating in the general population is 12%, but individuals with ADHD may face greater risks.

Those with the AMD showed greater expression of miRNA-150 than those without, offering evidence that miRNA-150 could be used as a potential biomarker—and drug target—for AMD.

New research gives more evidence about when and how quickly neurological deterioration occurs in patients with HIV.

Response to ketamine treatment in mood disorders may be better predicted based on baseline cognitive impairment.

It can’t replace two-step laboratory testing, but it is a suitable alternative as a diagnostic tool.

Fingolimod was first approved for adult patients with the relapsing form of MS, and was granted Priority Review and Breakthrough Therapy designation in December.

Late Friday afternoon, Novartis announced that the U.S. FDA approved fingolimod (Gilenya) to treat relapsing multiple sclerosis (MS) in children and adolescents age 10 years and older.

Heather Landau, M.D., Assistant Attending Physician at the Memorial Sloan Kettering Cancer Center, explains AL Amyloidosis. In this video, she describes in detail the clinical definition of the disease, what organs are affected, and early presentation.

Columbia University researchers believe CRISPR technology could also restore retinal function in the degenerative disease retinitis pigmentosa.

Results from a study recently published in The Lancet Neurology describe in detail a 16-year follow up of patients with hemophilia A who were treated with frequency-escalated prophylaxis.

AMO Pharma Limited announces the commencement of patient recruitment for an interventional study of AMO-01, an investigational Ras-ERK pathway inhibitor for the treatment of Phelan-McDermid syndrome (PMS).

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.