January 23rd 2024
Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
December 26th 2023
FDA Approves Treatment for Huntington's Disease
April 5th 2017Patients with chorea associated with Huntington’s disease have the first new treatment in nearly a decade. The FDA has approved Teva Pharmaceutical Industries Ltd’s Austedo (deutetrabenazine) for the treatment of chorea, which affects nearly 90% of patients with Huntington's.
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Analyzing the Near-Term Pipeline for Specialty Drugs
March 28th 2017The Academy of Managed Care Pharmacy Annual Meeting kicked off on March 28, 2017, in Denver, Colorado, with a look at the specialty pharmaceutical pipeline with Aimee Tharaldson, PharmD, senior clinical consultant for emerging therapeutics at Express Scripts.
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Dr Shaji Kumar: Advances and Opportunities in Treating Multiple Myeloma
March 25th 2017Despite the abundance of new drugs that have been approved recently and the promising clinical trials of other novel therapies, multiple myeloma remains incurable, explained Shaji Kumar, MD, professor of medicine at the Mayo Clinic. However, he believes that with better options for early intervention and personalized therapies, researchers are on a path to curing the disease.
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FDA Approves First Treatment for Rare Skin Cancer, Merkel Cell Carcinoma
March 23rd 2017The first treatment for Merkel cell carcinoma, a rare form of skin cancer, has been approved to treat adults and pediatric patients 12 years or older. The drug is also under consideration through Priority Review for treatment of patients with urothelial carcinoma.
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A Framework for Assessing the Value of Rare Disease Treatments
March 16th 2017Difficulty in making pricing and reimbursement decisions for rare diseases has prevented patients from having universal access to new medicines despite increased investment in their research and development. In Europe, a group has proposed 9 principles to guide value assessment, pricing, and funding.
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Cystic Fibrosis Survival in US Lags 10 Years Behind Canada, Researchers Find
March 14th 2017A concerning new study has found a significant gap in survival for cystic fibrosis patients in the United States and Canada. The 10-year advantage observed in Canadian patients could partially be explained by disparities in the countries’ healthcare systems, the researchers suggested.
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Raising Awareness of Rare Diseases and the Need for More Research
February 28th 2017The National Organization for Rare Disorders has announced a new, yearlong campaign to raise public awareness of rare diseases. “Do Your Share for Rare” shines a light on people living with a rare disease and provides an opportunity for them to share their story.
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Survey Finds Varied Views on Value of Multiple Sclerosis Therapies
February 21st 2017Population-level judgements on the value of therapies to treat multiple sclerosis don’t adequately depict the differences between how providers and payers view value compared with patients and how patients view value different from one another.
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Experimental Treatment Induces Remission in Multiple Sclerosis
February 16th 2017A new treatment option using stem cell transplants was shown to induce sustained remission of relapsing-remitting multiple sclerosis (MS). More than two-thirds of participants had no signs of progression of disability, relapse of MS symptoms, or new brain lesions after 5 years.
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Encouraging Results for New Strategy to Treat Juvenile Batten Disease
February 6th 2017A treatment for juvenile Batten disease has been tested in a mouse model and the results have shown reduced in symptoms and improved longevity. The results of the study were published in Nature Communications.
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Analysis Finds Alemtuzumab Most Cost-Effective Treatment for Multiple Sclerosis
February 2nd 2017A review of 15 disease-modifying therapies (DMTs) for the treatment of relapsing-remitting and primary-progressive multiple sclerosis (MS) has found that prices for most of these drugs are not well-aligned with added value for patients.
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First Trial With Universal CAR-T Treatment in Pediatric Leukemia Reports Success
January 26th 2017“Off-the-shelf” chimeric antigen receptor (CAR)-T cells, also known as universal donor cells, were used in 2 young infants with relapsed, refractory acute lymphoblastic leukemia resulted in molecular remission in 28 days in both infants.
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New Target for Treating Glioblastoma May Increase Survival Time
January 26th 2017Researchers from the Peter O’Donnell Jr. Brain Institute and Harold C. Simmons Comprehensive Cancer Center at the University of Texas Southwestern may have found the ultimate drivers of glioblastoma cell proliferation.
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