Working to Improve Gene Therapy for Adrenoleukodystrophy and Other Rare Conditions

Rare disease research is often underrepresented in the field of science. However, recent developments have made researchers more interested in studying this group of diseases. For instance, investments in gene…

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A Study Will Look at the Effects of an Experimental Drug on Childhood Cerebral ALD
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A Study Will Look at the Effects of an Experimental Drug on Childhood Cerebral ALD

A Phase 1/2 clinical trial of the experimental drug NV1205 in patients with childhood cerebral adrenoleukodystrophy (CCALD) has been initiated, announced NeuroVia Inc. The source article can be found here,…

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Is There a Genetic Condition in Your Family?
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Is There a Genetic Condition in Your Family?

Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…

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North Carolina Has Added Three More Conditions to Their Newborn Screening Program
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North Carolina Has Added Three More Conditions to Their Newborn Screening Program

Lawmakers in the U.S. state of North Carolina have expanded the state’s newborn screening testing to include three more conditions. These are Pompe disease, Mucopolysaccharidosis type 1 (MPS 1), and…

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An Experimental Gene Therapy for Cerebral ALD Has Been Granted Breakthrough Therapy Designation by the FDA
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An Experimental Gene Therapy for Cerebral ALD Has Been Granted Breakthrough Therapy Designation by the FDA

The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to the experimental drug Lenti-D, which is being produced by the gene therapy company Bluebird bio to treat cerebral…

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A Treatment for Inherited Metabolic Disorders is Being Trialled in Patients
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A Treatment for Inherited Metabolic Disorders is Being Trialled in Patients

A new treatment under development has produced promising results in patients with blood cancers and is now being tested in inherited metabolic disorders, reports Financial Buzz. Inherited metabolic disorders are…

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Treatment Development Getting Close for Patients with Form of Adrenoleukodystrophy
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Treatment Development Getting Close for Patients with Form of Adrenoleukodystrophy

Minoryx Therapeutics is on their way to making strides for patients with adrenomyeloneuropathy, also known as AMN, reports Labiotech. They just started their phase II initiative to test patients for…

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Editor’s Choice: Treatments, Methods and Strategies in Coping with Rare Diseases

Happy August Patient Worthy Peeps! This week, we have some awesome posts to keep you up-to-date on some of the latest rare disease developments. There's news on a hopeful possible…

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