Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders
source: pixabay.com

Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders

  According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…

Continue Reading Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders
A New Pediatric Precision Medicine Center is Opening in Utah
source: pixabay.com

A New Pediatric Precision Medicine Center is Opening in Utah

A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…

Continue Reading A New Pediatric Precision Medicine Center is Opening in Utah
New Trial Data Bodes Well for Experimental Cerebral Adrenoleukodystrophy Gene Therapy
skeeze / Pixabay

New Trial Data Bodes Well for Experimental Cerebral Adrenoleukodystrophy Gene Therapy

According to a story from pmlive.com, the gene therapy company Bluebird Bio's latest recently released results from the company's phase 2/3 clinical trial demonstrates further encouraging data for its Lenti-D…

Continue Reading New Trial Data Bodes Well for Experimental Cerebral Adrenoleukodystrophy Gene Therapy
Results From a Study of Gene Therapy for Cerebral Adrenoleukodystrophy Appear Positive
mcmurryjulie / Pixabay

Results From a Study of Gene Therapy for Cerebral Adrenoleukodystrophy Appear Positive

According to a story from PharmaTimes, the gene therapy company Bluebird bio has released updated results from a clinical study of its Lenti-D gene therapy as a treatment for patients…

Continue Reading Results From a Study of Gene Therapy for Cerebral Adrenoleukodystrophy Appear Positive
ICYMI: First Patient is Dosed in Phase 2 Friedreich’s Ataxia Trial
HeungSoon / Pixabay

ICYMI: First Patient is Dosed in Phase 2 Friedreich’s Ataxia Trial

According to a story from Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This trial…

Continue Reading ICYMI: First Patient is Dosed in Phase 2 Friedreich’s Ataxia Trial

Very First Patient Dosed with Investigational Therapy for Friedreich’s Ataxia in Phase 2 Clinical Trial

Friedreich's Ataxia Friedreich's ataxia (FRDA) is a rare disease which is caused by a frataxin deficiency. This deficiency results in the degeneration of nerves within the spinal cord. This nerve…

Continue Reading Very First Patient Dosed with Investigational Therapy for Friedreich’s Ataxia in Phase 2 Clinical Trial

Study Reveals Slow Progression of X-Linked Adrenoleukodystrophy in Women

According to a study published in the Orphanet Journal of Rare Diseases, a study that was intended to identify a new approach to monitoring the progression of x-linked adrenoleukodystrophy in women…

Continue Reading Study Reveals Slow Progression of X-Linked Adrenoleukodystrophy in Women
‘Mountain Man’ Maintains Work Out Regimen with ALD: A Wall Street Journal Profile 
Source: Pixabay.com

‘Mountain Man’ Maintains Work Out Regimen with ALD: A Wall Street Journal Profile 

Whose ready for some Monday motivation to get your week started? Readers of the Wall Street Journal might be familiar with their "What's Your Workout?" series, profiling someone's fitness and…

Continue Reading ‘Mountain Man’ Maintains Work Out Regimen with ALD: A Wall Street Journal Profile 

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…

Continue Reading Working to Improve Gene Therapy for Adrenoleukodystrophy and Other Rare Conditions
A Study Will Look at the Effects of an Experimental Drug on Childhood Cerebral ALD
Source: Pixabay.com

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,…

Continue Reading A Study Will Look at the Effects of an Experimental Drug on Childhood Cerebral ALD
Is There a Genetic Condition in Your Family?
Thor_Deichmann / Pixabay

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…

Continue Reading Is There a Genetic Condition in Your Family?
Jon’s Adrenoleukodystrophy Journey
Source: https://pixabay.com/en/forking-road-split-road-travel-839830/

Jon’s Adrenoleukodystrophy Journey

I met Jon at the 2018 United Leukodystrophy Foundation Conference and had the privilege of learning his and his family's story. Jon was born in Wisconsin in 1992, joining the…

Continue Reading Jon’s Adrenoleukodystrophy Journey
North Carolina Has Added Three More Conditions to Their Newborn Screening Program
Source: Pixabay

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…

Continue Reading North Carolina Has Added Three More Conditions to Their Newborn Screening Program
An Experimental Gene Therapy for Cerebral ALD Has Been Granted Breakthrough Therapy Designation by the FDA
Source: Pixabay

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…

Continue Reading An Experimental Gene Therapy for Cerebral ALD Has Been Granted Breakthrough Therapy Designation by the FDA
Understanding the ‘Building Blocks’ of ALD Patient Care
Source: Pixabay

Understanding the ‘Building Blocks’ of ALD Patient Care

Patient Worthy recently attended ALD Life’s Community Weekend 2018, which brought together people and families with Adrenoleukodystrophy (ALD) and Adrenomyeloneuropathy (AMN), and scientists, researchers, and doctors. One of the speakers…

Continue Reading Understanding the ‘Building Blocks’ of ALD Patient Care

This Man is Speaking Out About What it’s Really Like to Live With ALD

Michael Conway, 51, was diagnosed with Adrenoleukodystrophy (ALD) two years ago. Since then, he has been raising awareness for the rare and potentially life-shortening condition. He wants to let people…

Continue Reading This Man is Speaking Out About What it’s Really Like to Live With ALD
A Treatment for Inherited Metabolic Disorders is Being Trialled in Patients
Source: Pixabay

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…

Continue Reading A Treatment for Inherited Metabolic Disorders is Being Trialled in Patients
Close Menu