Finding a Treatment for ALS — Will Gene Editing Cut It?

 The Angel Fund supports the research of Dr. Robert H. Brown, Jr., world renowned ALS researcher. Dr. Brown is the Chair and professor of Neurology at UMass Medical School. Dr. Robert H. Brown, Jr. and the UMass Gene Therapy Center team are working on this exciting cutting-edge research using gene therapy to silence ALS genes.

Watch what Dr. Brown has to say about the progress of ALS research.

May , 2018
Finding a Treatment for ALS — Will Gene Editing Cut It?
Ammar Al‑Chalabi, F.R.C.P., Ph.D., and Robert H. Brown, Jr., M.D., D.Phil

A key challenge in human medical genetics is developing the ability to suppress the expression of mutant genes that cause diseases that are transmitted as dominant traits. This is particularly true for the neurodegenerative disorders; many such disorders have dominantly inherited genetic forms and are not reversible with current therapies. One example is amyotrophic lateral sclerosis (ALS): approximately 15% of cases can currently be attributed to dominant, high-penetrance gene variants,1 and many more ALS-causing genes probably remain to be discovered.

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Great news for ALS patients – FDA approves treatment for new drug !

Radicava (edaravone) has been approved by the FDA and will be available to treat ALS patients. MT Pharma America has been granted to begin marketing edaravone as a treatment for ALS. This is the first ALS treatment to be approved since 1995!!




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