Rare Disease

The FDA approved Spark Therapeutics Inc.’s voretigene neparvovec-rzyl (Luxturna), the first gene therapy for inherited vision loss caused by faulty gene mutations.

In a session at the 59th American Society of Hematology Annual Meeting and Exposition in Atlanta, Georgia, Brian Curtis, PhD, of the BloodCenter of Wisconsin Blood Research Institute, highlighted drugs other than chemotherapy that may cause neutropenia in patients.

Including the modified brentuximab antibody in the treatment regimen improved modified progression-free survival by 5%, although the rates of neutropenia and infections were higher in patients administered the brentuximab-containing regimen.

Is transfusion dependence a barrier to hospice utilization among older patients with leukemia who are enrolled in Medicare?

Infantile spasms are hard to diagnose, which can lead to treatment delays, according to the study authors.

Mantle cell lymphoma is an aggressive type of non-Hodgkin lymphoma. Acalabrutinib (Calquence) is a kinase inhibitor that works by blocking an enzyme the cancer needs to multiply and spread.

Although there is no scientific evidence that a specific diet reduces risk of infection in patients experiencing neutropenia, it is a common strategy in cancer care. Research presented at the 42nd Annual Congress of the Oncology Nursing Society determined that the popular neutropenic diet can be eliminated from practice.

Before treating certain patients, Daniel J. DeAngelo, MD, JD, of Harvard Medical School and the Dana-Farber Cancer Institute, will council them on the risk of developing neutropenia, which can result in hospitalization or even an intensive care stay.

After examining over 1000 cases of high-grade gliomas in children and young adults, researchers have split these rare brain tumors into at least 10 different subtypes, which could help clinicians choose and deliver more effective treatments.

FDA Commissioner Scott Gottlieb, MD, announced the agency’s progress in streamlining the orphan drug review process and outlined plans to close a loophole currently hindering pediatric drug research.

Bristol-Myers Squibb announced that the FDA has placed a clinical hold on 3 combination trials evaluating its programmed death-1 inhibitor nivolumab (Opdivo) in patients with relapsed/refractory multiple myeloma.

The assumption in the United States is that once someone with cancer is cured, done with treatment, and healthy, that they are now okay. However, the reality is that survivors contend with lingering challenges that aren’t visible and make it difficult for them to ask for help or admit they need it.

A look at some of the major developments in the chimeric antigen receptor T (CAR-T) cell space.

A gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB), a rare skin disease that causes the skin to be very fragile and blister easily, has been granted Breakthrough Therapy designation status by the FDA.

The FDA has approved the first chimeric antigen receptor-T (CAR-T) treatment, tisagenlecleucel (Kymriah), for the treatment of B-cell precursor acute lymphoblastic leukemia in certain pediatric and young adult patients. The therapy represents a new frontier in cancer care.

Chimeric antigen receptor (CAR)-T cells can re-expand in a lymphoma patient months after the initial infusion and can also be active against the patient’s cancer, according to a new study.

The FDA has approved Besponsa (inotuzumab ozogamicin) for treating adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

Patient preferences for multiple sclerosis therapies are important to take into account and are increasingly so as alternatives to traditional injection therapies are developed to address adherence issues.

The Institute for Clinical and Economic Review (ICER) has released a draft version of their scoping document that will compare the clinical effectiveness and value of 2 chimeric antigen receptor (CAR)-T cell treatments being reviewed by the FDA.

MimiVax announced that its immunotherapy vaccine SurVaxM, which is being tested in clinical trials for glioblastoma, has been designated an orphan drug by the FDA.

The results of a phase 2 study that explored the effects of luspatercept in patients with lower-risk myelodysplastic syndromes (MDS) were presented at the 22nd Congress of the European Hematology Association by Acceleron Pharma Inc. The company is developing the drug with Celgene.

Amidst news of Senator John McCain’s glioblastoma diagnosis, a recent discussion between 2 neuro-oncology researchers explored the reasons why this aggressive brain tumor is so difficult to manage, and outlined the ongoing investigations into possible therapies.

Patients with congenital heart defects, such as Marfan syndrome (MFS), are experiencing longer life expectancies due to new advances in medicine-resulting in a greater need for medical rehabilitation.