World desk:

Europe’s medicines watchdog gave the

green light Friday to a new treatment for a rare form of the
neurodegenerative disease ALS, saying it should reduce the symptoms of the
deadly illness.

Amyotrophic lateral sclerosis, sometimes called Lou Gehrig’s disease after
the famous baseball player, devastates nerve cells in the brain and spinal
cord. The mean survival time with ALS is two to five years.

Sold under the brand name “Qalsody”, the medicine toferson is aimed at adults
suffering from ALS as the result of a genetic mutation, the European
Medicines Agency said.

“The exact causes of ALS are unknown but are believed to include genetic and
environmental factors,” the Amsterdam-based EMA said.

“In approximately 2.0 percent of people living with ALS, the condition is
caused by a genetic mutation (change) that leads to the production of
defective SOD1 enzymes, causing nerve cells to die,” it said.

Qalsody reduces the production of the SOD1 protein and is therefore “expected
to improve the symptoms of ALS.”

To date, there is no effective neuroprotective treatment for all ALS
patients.

Despite the progress made in recent years, the disease currently remains
incurable.

There is only one other treatment for ALS, called “Riluzole”, authorised in
the EU.

Patients are also offered supportive treatment to relieve the symptoms of the
disease, such as physical, occupational or speech therapy and breathing
support.

“There is a large unmet medical need for effective therapies that preserve
muscle function and prolong the life of patients with ALS,” the EMA said.

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