Genetics in Disease
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- Cancer
- Investigating Packaged CAR T-Cells For Early Childhood Eye Cancer
- For some children with the rare eye cancer retinoblastoma a type of immunotherapy known as CAR T-cell therapy could be an effective treatment.
- 2068 0
- Weight and Obesity
- 1st Weight management treatment for people with certain rare genetic conditions approved
- People with obesity due to POMC, PCSK1 or LEPR deficiency become severely obese at a young age. This is the first FDA approved treatment for these conditions
- 1805 0
- Industry News
- FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies
- “Hutchinson-Gilford progeria syndrome and progeroid laminopathies are rare genetic diseases that cause premature aging and death and have a debilitating effect"
- 1696 0
- Industry News
- FDA Approves First Drug to Treat Rare Metabolic Disorder
- Approval is for primary hyperoxaluria type 1, which causes recurrent kidney stones and loss of kidney function.
- 1439 0
-
- Cancer
- Investigating Packaged CAR T-Cells For Early Childhood Eye Cancer
- For some children with the rare eye cancer retinoblastoma a type of immunotherapy known as CAR T-cell therapy could be an effective treatment.
- 2068 0
-
- Weight and Obesity
- 1st Weight management treatment for people with certain rare genetic conditions approved
- People with obesity due to POMC, PCSK1 or LEPR deficiency become severely obese at a young age. This is the first FDA approved treatment for these conditions
- 1805 0
-
- Industry News
- FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies
- “Hutchinson-Gilford progeria syndrome and progeroid laminopathies are rare genetic diseases that cause premature aging and death and have a debilitating effect"
- 1696 0
-
- Industry News
- FDA Approves First Drug to Treat Rare Metabolic Disorder
- Approval is for primary hyperoxaluria type 1, which causes recurrent kidney stones and loss of kidney function.
- 1439 0
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