Tag Archive for: Rare disease

The specialized region of the muscle where the motor neuron communicates the activation signal is at the neuromuscular junction. This is a specialized synapse between a nerve and muscle. In neuromuscular diseases there are deficits in both the motor neuron side (pre-synaptic) and on the muscle side (post-synaptic). © NDM Pharma
Rare disease

NDM Pharma provides POC for NMD670

Researchers at NDM Pharma A/S and Aarhus University have demonstrated that their oral CIC-1 blocker NMD670 restores crosstalk between neurons and muscles in Myasthenia gravis.

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© SynaptixBio Ltd
rare diseases

SynaptixBio Ltd gets FDA ODD for leukodystrophy

Rare disease specialist SynaptixBio Ltd has got its second FDA orphan drug designation for a subtype of TUBB4A leukodystrophy.

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