Raredr

Genetic

Genetic diseases can be rare, too. For resources on genetic mutations and how they pertain to rare diseases, turn to Rare Disease Report.
RDR Editorial: Deciding the Fate of Charlie Gard
On July 25, the Judge will decide the fate of Charlie Gard. What he decides may lean towards the hippocratic oath: First, do no harm.
A European team of scientists discovered what they claim are encouraging early signs for a potential Huntington’s disease (HD) treatment.
Intracerebral gene therapy infusions in 4 children with Sanfilippo type B led to sustained enzyme production & improved cognitive functions.
The safe position would have been to decline the request for fear of liability, error, harm to the unborn, to the parents. We took the case.
Team NeuroGate will be competing in the Microsoft Imagine Cup finals with their work that recognizes neurodegenerative disorders like HD and Parkinson’s.
Three different groups from the United Kingdom are staying active this week in their efforts to raise awareness of and funds for Huntington’s Disease research.
If their DMD drug gets approved, Capricor Therapeutics would receive a Priority Review Voucher that is worth millions of dollars.
The Huntington’s Disease Network DataBase (HDNetDB) will potentially reduce the complexity by allowing scientists to freely share data.
Allena announced that the FDA has granted Orphan Drug Designation to its compound, ALLN-177, for the treatment of primary hyperoxaluria.
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