Adrenoleukodystrophy (ALD) Therapeutics Pipeline Analysis by Therapeutics, Diagnostics, Patient, Drugs Policy and Regulatory Landscape

Adrenoleukodystrophy (ALD) Therapeutics Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations, and Other Developments published by Pharma Proff, ALD currently exhibits a proliferating pipeline with 10 therapeutic candidates.

ALD Pipeline Insights

ALD is a X-linked genetic disease which falls under the family of leukodystrophies disorder. The disease is monogenetically inherited from parents to offspring, with 100% penetrance in men and 65% in heterozygous women. It is a multi-system disorder which prominently affects both central and peripheral nervous system, resulting in blindness, seizures and hyperactivity.

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The pathophysiologic condition of disease includes the accumulation of saturated very long chain fatty acids (VLCFA) in serum and tissues of central nervous system, which is a major factor for immune system abnormality and demyelination of neurons. It mainly affects nervous system and adrenal glands and causes various adrenal and neurological problems, such as leg stiffness, muscles spasms and weakness, urinary problems, and sexual dysfunction.

Browse report overview with detailed TOC on "Adrenoleukodystrophy (ALD) Therapeutics Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations, and Other Developments" at: https://www.pharmaproff.com/report/adrenoleukodystrophy

Insights into Pipeline Segments

According to the research, many drugs acting as a therapeutic against ALD are administered orally. It has been found that oral route of administration is easy to use, non-invasive, and ensures high level of patient satisfaction.

It has been observed that regulatory bodies are granting designations to the drugs in ALD pipeline, in order to pace up the development process. For instance, bluebird bio Inc. ‘s drug candidate, Lenti-D, received Orphan Drug Designation by the USFDA and EMA in 2012; and Breakthrough Therapy Designation by the USFDA in 2018. Similarly, Minoryx Therapeutics S.L. drug, MIN-102, received Orphan Drug Designation by the USFDA in 2017.

Some of the key players involved in the development of ALD are Poxel SA, bluebird bio Inc., Minoryx Therapeutics SL, NeuroVia Inc., SOM Innovation Biotech SL, Viking Therapeutics Inc., Orpheris Inc., MedDay Pharmaceuticals, Applied Genetic Technologies Corporation (AGTC), and Neuralgene.