Rare Disease

Study Identifies Stressors Experienced by Patients Following Allo-HSCT

September 18th 2020, 8:00pm

Article

Researchers assess the psychosocial impact of allogeneic hematopoietic stem cell transplantation, particularly how preparing patients can assist in guiding health care providers in offering better care.

Researchers Highlight Need for Disease Registries Independent of Pharmaceutical Industry

September 11th 2020, 8:30pm

Article

As rare disease registries to assess orphan drugs are mainly established by the pharmaceutical industry, researchers argued post-marketing registries, functioning as marketing tools, have an underestimated influence on clinical practice.

What We’re Reading: Sanofi, GSK Begin Vaccine Trial; Steroids Effective in Treating COVID-19; ALS Treatment Breakthrough

September 3rd 2020, 2:00pm

Article

Sanofi and GSK begin human trials for their COVID-19 vaccine; steroids prove effective in severe COVID-19 cases; a drug combination poses a breakthrough in ALS treatment.

Ruxolitinib May Be Another Option for Children With Steroid-Refractory aGVHD

September 2nd 2020, 7:45pm

Article

The results, indicating that ruxolitinib may be a promising option for children with steroid refractory advanced graft-versus-host disease (aGVHD), would need confirmation from a larger trial.

Report Describes Use of Ruxolitinib Over the Long-Term for Young Patient

August 29th 2020, 10:45am

Article

Researchers discuss the case of a 32-year-old patient who is being treated with ruxolitinib over a period of years to treat steroid refractory graft-versus-host disease.

Long-Term Analysis Highlights Survival Improvements for Grade 3 aGvHD

August 28th 2020, 5:15pm

Article

A recent report illustrates how advances in allogeneic hematopoietic stem cell transplantation (HSCT) have impacted the outcomes of children with severe acute graft-versus-host-disease.

Ruxolitinib Combo Shows Promising Results in Patients With Steroid-Resistant aGVHD

August 19th 2020, 10:00am

Article

Ruxolitinib plus etanercept showed positive results for the treatment of steroid-refractory severe acute graft-versus-host disease.

FDA Approves Third Drug for NMOSD

August 17th 2020, 7:12pm

Article

Satralizumab is the third drug to be approved by the FDA for neuromyelitis optica spectrum disorder (NMOSD).

Report Details First Noted Pediatric Case of Autoimmune Myelofibrosis

August 14th 2020, 3:15pm

Article

An adolescent boy went to his physician with a headache that got worse with physical activity. Testing showed autoimmune myelofibrosis.

Real-World Study Backs Long-term Use of Ruxolitinib in Myelofibrosis

August 12th 2020, 4:00am

Article

A real-world study showed that ruxolitinib is a safe treatment for patients with myelofibrosis.