Rosalyn Blumenthal, PhD

Rosalyn Blumenthal has a PhD in Biomedical Science from The City University of New York with a specialization in physiology and biophysics. She is a trained researcher, educator, medical writer, and medical strategist. Dr. Blumenthal has more than 30 years of experience developing medical content and is passionate about making a difference for patients with rare diseases.

All articles by Rosalyn Blumenthal, PhD

Clinical trial

Microsimulation Model Estimates Effectiveness of RRMS Treatment Sequences

A microsimulation model published in Value in Health estimated the effectiveness and cost-effectiveness of 360 treatment sequences in terms of health outcomes and societal costs, beginning with first-line therapies in relapsed-remitting multiple sclerosis (RRMS). The decision-analytic modeling conducted in the Netherlands integrated different data sources and included information on “disease progression, disease-modifying treatment efficacy, clinical…

Clinical Trial

Potential IPF Treatment Cudetaxestat Posts Positive Phase 1 Results

Positive topline data from a phase 1 clinical study (NCT04814498) of cudetaxestat (BLD-0409) have been presented by Blade Therapeutics, a South San Francisco biopharmaceutical company. The study evaluated the effect of cudetaxestat on the pharmacokinetics of a combination of probe substrates for CYP450 enzymes. No alteration of plasma levels of substrates for cytochromes 3A4, 2B6,…

Ataluren Delays Respiratory Decline and Loss of Ambulation in Patients With Nonsense Mutation DMD

A long-term phase 3 study found that ataluren plus standard of care (SoC) delays progression of nonsense mutation Duchenne muscular dystrophy (nmDMD) and benefits ambulatory and nonambulatory patients. Ataluren is an oral therapy for patients with nmDMD that enables ribosomes to read through a premature stop codon in mRNA, allowing translation to continue and resulting…

joint degeneration

Improvements Seen in Perioperative Management of Adults With Hemophilia

A Canadian multicenter 3-decade retrospective cohort review of adults with hemophilia, published in Haemophilia, reported a decrease in total knee arthroplasties (TKA) and an increase in the rate of ankle arthrodesis over time. In addition, length of stay (LOS) and factor utilization decreased, reflecting improvements in perioperative management. Surgical intervention is often needed to address…

Eptacog Beta as a Potential New Therapeutic Option for Hemophilia

Eptacog beta is a well-tolerated and effective bypassing agent (BPA) for major and minor surgical procedures in pediatric and adult patients with hemophilia A or B with inhibitors (PwHABI), according to results from the phase 3 PRESEPT trial published in Hemophilia. The BPA human recombinant factor VIIa (eptacog beta) was prospectively evaluated in 12 individuals,…

plant-based diet

Demographic and Clinical Characteristics of Adherence to Selected MS-Specific Diets

A new study published in Multiple Sclerosis and Related Disorders reported that 48.8% of participants in a large international cohort with multiple sclerosis (MS) followed an MS-specific diet program for at least 12 months. The most common programs in this cohort were Overcoming MS (38.1%), Swank (6.3%), and Wahls (3.1%). Data were collected from 952…

A Potential Tool for Detecting Subtle Left Ventricular Systolic Dysfunction in DMD

A new study published in the Egyptian Heart Journal reported that left ventricular (LV) global longitudinal strain (GLS) could be used to detect subtle LV systolic dysfunction in patients with Duchenne muscular dystrophy (DMD) and in female carriers before the onset of cardiac symptoms and before the decline in LV ejection fraction (EF). Earlier identification…

Tranexamic Acid Shows Benefits During Scoliosis Surgery in Patients With SMA

A new study published in the International Journal of Environmental Research and Public Health reported that perioperative IV tranexamic acid (TXA), a fibrinolysis inhibitor, can decrease intraoperative blood loss and crystalloid fluid volume overload during posterior spinal fusion surgery for scoliosis in patients with spinal muscular atrophy (SMA). These changes may allow earlier postoperative extubation…

doctor in surgery

Long-Term Survival of Patients With GISTs Similar With Endoscopic Resection and Surgery

A new study published in Surgical Endoscopy reported that local endoscopic resection and open, or laparoscopic, surgical resection resulted in similar long-term survival of American patients with gastric or colorectal gastrointestinal stromal tumors (GISTs).  This is the first large population-based study to compare endoscopic resection with surgical resection with a relatively long follow-up period. Addressing…

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