Conference Coverage
Conference Coverage
12/19/2024
Miranda Manier, BA
A specialized inpatient sickle cell disease (SCD) consult service led to improved pain management, shorter hospital stays and expanded use of individualized pain plans for patients with SCD.
12/19/2024
Research Summary
Research Summary
05/24/2024

Anthony Calabro, MA

Anthony Calabro, MA
Oxalate nephropathy is a commonly underrecognized cause of chronic kidney disease and end-stage kidney disease with multiple etiologies, including primary hyperoxaluria, enteric hyperoxaluria, or ingestion...
05/24/2024
RESEARCH SUMMARY
RESEARCH SUMMARY
05/22/2024

Anthony Calabro, MA

Anthony Calabro, MA
A recent cross-sectional, observational study compared the tau spread in people with Down syndrome and autosomal-dominant Alzheimer disease to identify tau-related differences between the two groups.
05/22/2024
research summary
research summary
05/20/2024
Leigh Precopio
Researchers examined the epidemiology of hydroxymethylbilane synthase (HMBS) gene defects and activity levels among patients with acute intermittent porphyria to better understand the role of HMBS activity...
05/20/2024
FDA Alert
FDA Alert
04/18/2024

Anthony Calabro, MA

Anthony Calabro, MA
The FDA approved givinostat, a nonsteroidal oral medication, for treating patients 6 years of age or older with Duchenne muscular dystrophy.
04/18/2024
FDA Alert
FDA Alert
03/19/2024

Jessica Ganga

Jessica Ganga
The FDA approved two gene therapies, the first of their kind, to treat patients aged 12 years and older with sickle cell disease.
03/19/2024
Research Summary
Research Summary
10/26/2023
Anthony Calabro, MA
Researchers set out to determine the impact of midostaurin in adult patients with FLT3-mutated acute myeloid leukemia.
10/26/2023
FDA ALERT
FDA ALERT
04/18/2023
Jessica Ganga
The FDA has approved a treatment for Friedreich ataxia after the results of randomized, placebo-controlled study indicate a strong effectiveness in treatment.
04/18/2023
Enzyme Replacement Therapy
Enzyme Replacement Therapy
03/17/2023
Jessica Ganga
Following a multicenter study, the FDA has approved the first enzyme replacement therapy in the United States to treat alpha-mannosidosis.
03/17/2023
Bleeding Disorders
Bleeding Disorders
11/28/2022
The first of its kind gene therapy treatment has been approved by the FDA for adults with Hemophilia B.
11/28/2022