x chromosome

Merck Serono, a Geneva-based subsidiary of Merck, has announced the launch of the EspeRare Foundation to investigate the compound rimeporide in Duchenne Muscular Dystophy, a rare but fatal paediatric disease.

The company will donate an initial funding of €2.8m to the nonprofit foundation, set up by three Merck Serono employees, and transfer the rights of rimeporade, which was previously developed for heart failure.

Sharon Terry, president and CEO of Genetic Alliance and an executive board member of the International Rare Disease Research Consortium, will join the organisation.

"The International Rare Disease Research Consortium has publicly and boldly declared that we, as a global community, will have 200 new drugs for rare diseases by 2020," Terry said in a statement.

"The EspeRare Foundation has a great potential to become an important component in the new system that must be built to achieve this goal. The current pharma model needs to be adapted, new pathways are being forged and the Foundation will be a catalyst and incubator for a new way of addressing rare diseases."

Duchenne Muscular Dystophy is a lethal genetic rare disease affecting one in every 3,600 males.

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By GlobalData

The disorder is caused by a mutation in the dystrophin gene, located on the human X chromosome, which is responsible for the structure and functioning of muscles.

While both sexes can carry the mutation, females rarely exhibit signs of the disease.

Image: Duchenne Muscular Dystophy is cause by mutations in the human X chromosome. Image courtesy of FreeDigitalPhotos.net.