Spinal Muscular Atrophy – Rays of Hopes!

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Some rays of hopes are shining on the horizon for those who are either suffering themselves or their beloved ones are suffering with spinal muscular atrophy, a hereditary disease which weakens muscles and snatches ability to move in children. Repligen, a leading company in the field of drug technologies and research, has performed trial of a drug on spinal muscular atrophy, named RG3039 and has told that the results from the Phase I study are encouraging.

RG3039 Trial

The blinded single dose ascending research revealed that RG 3039 was tolerated well with all administered doses, thus proving a dose-associated drug response which results in 90% stoppage of the target enzyme. It is expected that the results will help to set accurate dose regimens for upcoming studies, including probable efficiency researches in SMA patients as per the company.

Causes of Spinal Muscular Atrophy

Spinal muscular atrophy is believed to be caused by decreased quantities of a protein in nerve cells. It is called survival motor neuron protein or SMN. Scientists have found out more clues about the causes of the disease, according to which another protein named plastin 3 also has a major role in the occurrence of the disease. According to the studies, decrease in SMN causes decrease in plastin 3 and when plastin 3 is added to motor neurons the defects, seen when SMN is lowered, are rescued. This suggests that reduction in plastin 3 contribute to the manifestation of the disease. The disease affects 1 amongst 6,000 to 10,000 individuals and 1 amongst 40 people is carrier of this disease.

At present there is no definite cure for spinal muscular atrophy. And so, scientists are trying their best to find one. RG3039 is a drug still at a clinical stage. It is targeted on the key genetic decline of survival motor neuron protein which may possibly treat the biochemical decline caused by the reduced levels of the SMN.

Boon for SMA Patients

In 2009, RG3039 has been licensed from the non-profit organization for patient support named Families of Spinal Muscular Atrophy. It was tried in 32 healthy volunteers. It is the first clinical-stage medicine to treat reduced levels of survival motor neuron protein. If the trials for drug RG3039 are successful, it will act like a boon for the spinal muscular atrophy patients around the world.

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