New Marker Found to Track Disease Progression in Charcot-Marie-Tooth Disease Type 1a

A study conducted by researchers from Aix-Marseille University in France has revealed a new way to track disease progression of Charcot-Marie-Tooth disease type 1a (CMT1a)- changes to the nerves within the thigh.…

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$950,000 Raised to Fund NeuroMotor Pen, A Diagnostic Tool for Neurological Disorders
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$950,000 Raised to Fund NeuroMotor Pen, A Diagnostic Tool for Neurological Disorders

Every day, the world becomes increasingly technological. With this comes advances in many realms, from healthcare to education. Recently, says Parkinson's News, medical technology company Manus Neurodynamica received £750,000 (approx. $950,000)…

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Positive Results Announced for a Phase 1 Trial Targeting Root of Chronic Urticarias
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Positive Results Announced for a Phase 1 Trial Targeting Root of Chronic Urticarias

Celldex has just released positive results from a Phase 1 trial of an investigative therapy which could benefit patients living with chronic urticarias. This disease can result in decreased energy,…

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US Veterans with Pulmonary Hypertension Are Not Treated with Exercise Rehabilitation

According to Healio, US veterans affected by pulmonary arterial hypertension are typically not treated with exercise rehabilitation, despite the fact that many medical professionals believe that utilizing exercise rehabilitation in…

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This Woman is the First Ever Patient to Have Both aHUS and Cobalamin C Deficiency

When the 19-year-old French woman first visited the hospital, she was extremely lethargic. She displayed neurological symptoms. Upon kidney failure, the patient was admitted into the ICU. But soon, doctors…

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Expressive Language Sampling May Lead to Treatment Options for Fragile X Syndrome
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Expressive Language Sampling May Lead to Treatment Options for Fragile X Syndrome

By Danielle Bradshaw from In The Cloud Copy Expressive language sampling, or ELS, could potentially help researchers better measure how well treatments work for patients with fragile X syndrome or…

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Experimental Treatment for Neuroblastoma Earns Rare Pediatric Disease Designation
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Experimental Treatment for Neuroblastoma Earns Rare Pediatric Disease Designation

According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals has recently announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to its…

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Study: Nuplazid as a Treatment for Psychosis in Parkinson’s Disease Patients
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Study: Nuplazid as a Treatment for Psychosis in Parkinson’s Disease Patients

Acadia Pharmaceuticals recently released the results of their Phase 3 trial of Nuplazid, a treatment for the psychosis that Parkinson's disease patients experience. This medication has been approved since 2016,…

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Patients with Lesch-Nyhan Syndrome Have Biochemically Altered Skin, Urine, and Spinal Fluid
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Patients with Lesch-Nyhan Syndrome Have Biochemically Altered Skin, Urine, and Spinal Fluid

According to News Medical, a recent international study designed to understand Lesch-Nyhan syndrome and its potential treatments discovered something intriguing: biochemical changes in the skin, cerebrospinal fluid, and urine. Now,…

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Filmmaker with Muscular Dystrophy Wins Award for “Muscle Warriors” Documentary
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Filmmaker with Muscular Dystrophy Wins Award for “Muscle Warriors” Documentary

  According to Disability Horizons, an extremely talented filmmaker with muscular dystrophy recently won an Award of Recognition from The Best Shorts Film Competition. Stephanie Castelete-Tyrrell's film, entitled Muscle Warriors, documents…

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Consumers with Allergies, EoE Worry When FDA Relaxes Food Labeling Guidelines
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Consumers with Allergies, EoE Worry When FDA Relaxes Food Labeling Guidelines

  Stacey Saiontz is many things: a worker, a wife, a mother. But right now, she is concerned. As an allergy parent, or a parent whose child has severe food…

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NORD Announces Eleven New Grants for Rare Disease Research
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NORD Announces Eleven New Grants for Rare Disease Research

According to a story from PR Newswire, the National Organization for Rare Disorders (NORD) has recently awarded a total of eleven new grant awards that will help fund essential research…

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