Rare Disease

FDA Clears Thymus Tissue Product to Treat Congenital Athymia

October 11th 2021, 9:30pm


The product, called Rethymic, is composed of human allogeneic thymus tissue that is processed and cultured and then implanted into children born without a thymus to help reconstitute immunity.

Ruxolitinib Has Cost-effectiveness, Survival Benefits for Myelofibrosis vs Competitors

October 6th 2021, 1:00pm


Research presented at EHA2021 shows that the JAK 1/2 inhibitor ruxolitinib produced cost-effective benefits per quality-adjusted life-year and increased the overall survival rate for patients with myelofibrosis.

Management of Low-risk ET and PV Has Room for Improvement

October 3rd 2021, 2:00pm


Despite excellent prognoses in low-risk patients with essential thrombocytopenia (ET) and polycythemia vera (PV), knowledge gaps remain and novel, more tolerable therapies require reevaluation of treatment algorithms.

Pediatric AADC Deficiency Improves With New Gene Therapy

October 1st 2021, 9:00pm


This new analysis of 5-year data on a novel gene therapy from PTC Therapeutics shows both cognitive and motor function improvement in children with aromatic L-amino acid decarboxylase (AADC) deficiency, a rare central nervous system disorder.

Researchers Detail Case of Statin-Induced Necrotizing Myositis

September 26th 2021, 12:00pm


Immune-mediated necrotizing myopathy typically presents with progressive symmetrical proximal muscle weakness and myalgias, although some cases can present with respiratory and esophageal muscle weakness, as seen in this reported case study.

FDA Approves Ruxolitinib for Chronic GVHD

September 22nd 2021, 6:30pm


Chronic graft-vs-host disease (GVHD) is a condition that can occur after an allogeneic stem cell transplant in which the donated cells initiate an immune response and attack the transplant recipient’s organs.

Ruxolitinib Likely Safe, Effective in Chronic GVHD

September 21st 2021, 4:48pm


The study of 36 patients showed a response rate of 59% at 3 months, which increased to 62% at 6 months.

JAK Inhibitor Treatment in Myelofibrosis May Depend on Treatment Line, Risk of AEs

September 18th 2021, 6:33pm


The data offer novel insights, as comparable data on the drug class is scarce despite 2 JAK inhibitors being approved for use in myelofibrosis and others showing promise.

Managing Cerebrovascular Complications in Patients With MPN

September 18th 2021, 11:40am


Complications of note in patients with myeloproliferative neoplasm (MPNs) include ischemic stroke, intracerebral and subarachnoid hemorrhage, microbleeds, posterior reversible encephalopathy syndrome, and dural sinus and cerebral vein thrombosis.

Multistakeholder Panel Weighs In on Which Factors to Consider for Reimbursement of Orphan Drugs

September 11th 2021, 9:00pm


From the decision analysis came an agreement that reimbursement for an orphan drug should be based primarily on its effect on health-related quality of life, its effectiveness, and the availability of other treatment options.