The Results of this Study Could Improve Outcomes for Duchenne Muscular Dystrophy
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The Results of this Study Could Improve Outcomes for Duchenne Muscular Dystrophy

Last month, the pharmaceutical company Sarepta Therapeutics announced the long awaited results of a study that measured lung function in patients with Duchenne muscular dystrophy (DMD) after they took eteplirsen,…

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These Rare Disease Treatments Just Became a Lot More Affordable for Australians
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These Rare Disease Treatments Just Became a Lot More Affordable for Australians

The Australian government has announced that almost that new treatments worth $300 million are being added to the Pharmaceutical Benefits Scheme. This program provides subsidized medical treatments to citizens of…

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Medical Professionals Are Starting To Debate Clinical Trial Eligibility Standards
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Medical Professionals Are Starting To Debate Clinical Trial Eligibility Standards

According to an article from the Chicago Tribune, many medical specialists are coming to the realization that the eligibility criteria for clinical trials should be reassessed. The problem is that…

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Ministry of Health Focuses on Creating Funds for Families with Rare Diseases in Singapore
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Ministry of Health Focuses on Creating Funds for Families with Rare Diseases in Singapore

Out-of-pocket expenses for rare disease are astronomical, and the Ministry of Health (MOH), is taking time to find a more positive way to help benefit children and their families burdened…

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Albanian Father Caring For Wife With Multiple Sclerosis Threatened by Deportation
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Albanian Father Caring For Wife With Multiple Sclerosis Threatened by Deportation

Ded Rranxburgaj, 48, currently living in Southgate, Michigan is being threatened with deportation, although his wife suffering from multiple sclerosis, reported MSN News. While fighting the deportation from every angle, Ded…

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AveXis, Gene Therapy Development Company, Expands Program
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AveXis, Gene Therapy Development Company, Expands Program

The gene therapy development company, AveXis, Incorporation, just announced that they will be expanding their clinical trial development program for the potential gene therapy treatment: AVXS-101, reported Globe News Wire.…

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Parents Sue Hospital and Social Services Over Treatment Of Their 11-Year-Old Daughter with Rett Syndrome
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Parents Sue Hospital and Social Services Over Treatment Of Their 11-Year-Old Daughter with Rett Syndrome

The Driscoll family is left devastated and shocked after receiving a letter from social services that states the government could be taking their daughter, who is suffering from Rett syndrome,…

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