A rare genetic disorder discovered by UT Southwestern Medical Center researchers and their colleagues can cause brain damage ...
A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...
Study Finds on MSN
New blood test shows promise for faster ALS diagnosis
No more waiting months, or even over a year, for an ALS diagnosis? In A Nutshell A 46-gene blood test identified ALS patients ...
9monon MSN
Study links blood-brain barrier dysfunction and inflammation with Down syndrome regression disorder
A new study led by Jonathan D. Santoro, MD, Director of the Neuroimmunology Program at Children's Hospital Los Angeles, shows ...
Early detection and innovative therapies are crucial for managing rare blood disorders like VITT, ITP, and hemophilia. Marstacimab's approval offers a new subcutaneous treatment option for hemophilia ...
While Novo Nordisk has been operating in rare disease for some 45 years, the field has often taken a back seat to the Danish drugmaker’s bread-and-butter business developing treatments for diabetes ...
We found little to no difference in the number of women developing pre-eclampsia when they were given calcium during pregnancy, and we are uncertain about poor outcomes for mothers and babies. Most ...
3don MSN
Genetic overlap of 14 psychiatric disorders explains why patients often have multiple diagnoses
An international collective of researchers is delivering new insights into why having multiple psychiatric disorders is the ...
A new research study led by Jonathan D. Santoro, MD, Director of the Neuroimmunology Program at Children's Hospital Los Angeles, shows evidence of dysfunction of the blood-brain barrier and ...
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