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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

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

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

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
Genetics
Genetics
11/23/2022
The researchers set out to determine whether rare variations in certain genes contribute to the risk of developing sJIA.
11/23/2022
Adhesive Capsulitis
Adhesive Capsulitis
10/18/2022
Adhesive capsulitis, known as “frozen shoulder,” has a strong association with specific gene variants
10/18/2022
Medication
Medication
09/20/2022
The FDA approved an intravenous infusion medication to treat symptoms of this rare genetic disease that affects the liver, spleen, lung, and brain.
09/20/2022
genetics
genetics
12/13/2019
In a new study, researchers used the largest aggregate of genome-wide association studies data to identify novel risk loci and gained further insight into the causes of Parkinson disease.
12/13/2019