Rare Disease

Top 5 Most-Read Rare Disease Stories of 2020

December 26th 2020, 3:00pm

Article

The top 5 most-read stories of 2020 about rare diseases on AJMC.com focused mainly on new and noteworthy treatment options for a variety of conditions.

Dr Clarence Moore Discusses Patient Accessibility to Beta Thalassemia Treatments

December 21st 2020, 12:00pm

Video

Clarence Moore, PharmD, BCPS, BCOP, assistant professor at Shenandoah University in Ashburn, Virginia, discusses whether treatment methods for beta thalassemia are accessible to patients.

What We’re Reading: AstraZeneca to Buy Alexion; CDC Guidance on Vaccine, Allergy Concerns; First Vaccines Headed to States

December 14th 2020, 3:39pm

Article

AstraZeneca has agreed to acquire Alexion Pharmaceuticals for $39 billion; CDC issues coronavirus disease 2019 (COVID-19) vaccine guidance for those with a history of severe reactions to vaccines; 2.9 million COVID-19 vaccine doses began traveling this past weekend to all 50 states.

Dr Clarence Moore Discusses the Importance of Stem Cell Therapy, Heart Health in Beta Thalassemia

December 13th 2020, 11:30am

Video

Clarence Moore, PharmD, BCPS, BCOP, assistant professor at Shenandoah University in Ashburn, Virginia, discusses how stem cell therapy and heart health can help patients manage beta thalassemia.

What We’re Reading: VA Health Care Fight; High Court Sides With States on PBMs; Ezogabine and ALS

December 11th 2020, 5:09pm

Article

A funding dispute over how to account for a Veterans Affairs (VA) health care program is stalling deal to fund the government for another year; the Supreme Court rules in a case involving states and pharmacy benefit managers (PBMs); the antiepileptic drug ezogabine lowered the pathologic excitability of cortical and spinal motor neuron cells, which have a role in amyotrophic lateral sclerosis (ALS).

GWAS Identifies New Genetic Associations of Takayasu Arteritis

December 11th 2020, 4:00pm

Article

Researchers identified new genetic associations that can predict individuals’ susceptibility to Takayasu arteritis, a rare type of vasculitis.

Ruxolitinib Shows Efficacy in Patients With Steroid-Resistant, Dependent Chronic GVHD

December 6th 2020, 11:30am

Article

The new data provide hope that the therapy will prove effective for a patient group with limited options.

What We’re Reading: FDA Issues Warning Letter; UK Approves COVID-19 Vaccine; Flu Vaccine Safe

December 2nd 2020, 3:10pm

Article

Avazo-Healthcare warned by the FDA about its coronavirus disease 2019 (COVID-19) testing kits; Pfizer, BioNTech get first COVID-19 vaccine approval; risk for Guillan-Barré syndrome from flu vaccine deemed insignificant.

Pacritinib Shows Promise in Phase 2 Advanced Myelofibrosis Study

December 1st 2020, 8:15pm

Article

A new dose-finding study found a twice-daily 200 mg dose of the JAK2/IRAK1 inhibitor pacrinitib led to a spleen volume response in nearly 1 in 10 patients.

Dr Clarence Moore: Luspatercept Is a Positive Step Forward in Beta Thalassemia

November 27th 2020, 11:00am

Video

Clarence Moore, PharmD, BCPS, BCOP, assistant professor at Shenandoah University in Ashburn, Virginia, discusses a new treatment option for beta thalassemia.