New funding brings personalized CRISPR cures closer to children with rare diseases

	Rare Diseases
	The latest rare diseases news from News Medical

	New funding brings personalized CRISPR cures closer to children with rare diseases Personalized CRISPR cures for children born with rare genetic diseases are now a step closer to being more widely available.

	Experimental treatment reverses paralysis in child with rare mitochondrial disease The value of a recent biochemical discovery can be seen in the case of an 8-year-old boy. In August 2023 he was able to play typical sports, but by November he needed a wheelchair because of a rare disease that caused worsening paralysis.

			Oral drug MA-5 can improve heart and muscle problems in Barth syndrome Researchers at Tohoku University have discovered that an oral drug called MA-5 can improve both heart and muscle problems in Barth syndrome, a rare genetic disorder affecting 1 in 300,000 births worldwide with no current cure.

			Clinical trial examines whether Ambroxol can slow dementia in people with Parkinson's Dementia poses a major health challenge with no safe, affordable treatments to slow its progression.

			Researchers develop blood test panel for early Alzheimer’s detection Researchers at the Federal University of São Carlos (UFSCar) in the state of São Paulo, Brazil, are developing a panel of biomarkers for the early detection of Alzheimer's disease and for distinguishing it from other types of dementia using blood tests.

	In vivo base editing marks new era in personalized genetic medicine In vivo base editing enables precise, single-nucleotide changes to DNA without double-strand breaks. A recent application in CPS1 deficiency shows promise for personalized treatments in rare genetic disorders.

	New small molecule shows promise against cutaneous HPV infections Human papillomaviruses, or HPVs, can cause dangerous infections in the skin and mucous membranes and may lead to cancer.

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