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New report shows nearly 20 innovative medicines in development for sickle cell disease
Sickle cell disease (SCD) is the most common inherited blood disorder in the United States, afflicting nearly 100,000 Americans. Patients with SCD have sickle-shaped red blood cells, which have trouble moving through the blood vessels and thus cause blockages or slow blood flow and oxygen to parts of the body. SCD is life-threatening, due to potential complications from blocked blood vessels, which can include stroke, difficulty breathing, pulmonary hypertension and other organ damage.
Source: The Catalyst - April 2, 2019 Category: Pharmaceuticals Tags: Medicines in Development Research and Development Rare Diseases New Era of Medicine Source Type: news

Pelosi ’s radical plan would leave patients with sickle cell disease behind
Sickle cell disease (SCD) is the most commonly inherited genetic blood disorder, affecting nearly 100,000 children and adults in the United States. Three million Americans carry the sickle cell trait and it is nearly25 times more common in people of African American descent. The disorder, which can cause irregularly shaped blood cells to get stuck in narrow vessels, inflicts substantial pain on patients. The resulting reduced oxygen flow creates life-threatening complications such as difficulty breathing, chest pains, blood clots and stroke.
Source: The Catalyst - November 14, 2019 Category: Pharmaceuticals Authors: Tom Wilbur Tags: Research and Development Rare Diseases Pelosi Plan Source Type: news

Acute Flaccid Myelitis: A Clinical Review
Semin Neurol DOI: 10.1055/s-0040-1705123Acute flaccid myelitis (AFM) is an emerging disorder primarily affecting children that is characterized by acute flaccid paralysis accompanied by abnormalities of the spinal cord gray matter on magnetic resonance imaging. In most cases, prodromal fever or respiratory symptoms occur, followed by acute-onset flaccid limb weakness. Respiratory, axial, bulbar, facial, and extraocular muscles may also be affected. The clinical manifestations have been described as “polio-like,” due to striking similarities to cases of poliomyelitis. The primary site of injury in AFM is the anterior ho...
Source: Seminars in Neurology - March 5, 2020 Category: Neurology Authors: Murphy, Olwen C. Pardo, Carlos A. Tags: Review Article Source Type: research

When does life end? New organ donation strategy fuels debate
On a chilly holiday Monday in January 2020, a medical milestone passed largely unnoticed. In a New York City operating room, surgeons gently removed the heart from a 43-year-old man who had died and shuttled it steps away to a patient in desperate need of a new one. More than 3500 people in the United States receive a new heart each year. But this case was different—the first of its kind in the country. “It took us 6 months to prepare,” says Nader Moazami, surgical head of heart transplantation at New York University (NYU) Langone Health, where the operation took place. The run-up included oversight from an ethi...
Source: ScienceNOW - May 11, 2023 Category: Science Source Type: news