There may or may not be a connection between my 8-year-old son’s 3 a.m. confession that he knows he might be “going to heaven ...

X-linked adrenoleukodystrophy (X-ALD) is a debilitating disease caused by mutations in the ABCD1 gene. Approximately 30% of affected boys develop the progressive ...

It is either tragically funny or devastatingly sad that while a genetic counselor explained the random lunacy of my son’s ...

X-Linked Adrenoleukodystrophy (ALD) is a rare genetic disorder that breaks down myelin, the protective sheath that surrounds the brain and spinal cord. Early intervention and regular screenings ...

It’s a rare genetic disorder that impacts a small population, and due to limited research, much remains unknown about ...

From Adrenoleukodystrophy to Zollinger Ellison Syndrome, there are currently over 7,000 diseases identified as rare. In the U.S., the Orphan Drug Act of 1983 defines a disease as “rare” if it affects ...

And I predict that 2025 will be another momentous year for Summit. Summit already owned the license to market ivonescimab in ...

Subsequent biochemical and genetic analysis revealed that the family was in fact affected by the adrenomyeloneuropathy subtype of X-linked adrenoleukodystrophy. In the family described, both males and ...

Research analysts at Leerink Partnrs upped their Q4 2024 earnings estimates for shares of bluebird bio in a research report issued on Wednesday, January 22nd. Leerink Partnrs analyst M. Foroohar now ...

The New Mexico Attorney General and a state senator are calling on lawmakers to create new criminal and civil penalties for ...

Before we learned a genetic glitch had messed up his expiration date, handing his life over to a cruel disease called adrenoleukodystrophy. Without a bone marrow transplant, he will waste away as the ...