MDA Update

I first would like to thank everyone who helped to make this year’s MDA Drive

successful. Whether you played golf at our outing, sponsored a hole, purchased a Red Ticket

for our raffle, or helped with the selling of the tickets I appreciate your help. This year’s Red

Ticket Raffle winner for the Grand Prize of $500.00 was Steve Feldman out of Lawrence. We

also had two runners-up that received $60.00 Gift Cards for Applebee’s, and they were David

Pena from Wantagh North and Sal Governale out of East Setauket. Thanks to everyone’s

efforts we were able to donate $16,000.00 for the 2024 campaign.

The NALC has been a supporter and is the official charity for MDA since 1952. The

NALC has raised over 105 million dollars as of 2023. The money we raise goes towards many

different types of research and the MDA camps for children suffering from this debilitating

disease.

MDA Summer Camp is free for families of children and young adults between the ages

of 8-17 with neuromuscular diseases. The average cost for one child to go to MDA Summer

Camp is $3,000.00. The camp is made possible by generous donors and supporters like the

NALC and Branch 6000 membership. As someone who has volunteered at one of these camps,

it is something to see. The joy on these children’s faces is priceless. The following activities are

provided for the children to take part in:

● Swimming,

● Adaptive sports,

● Boating,

● Fishing,

● Music,

● Arts and Crafts,

● Dances,

● Talent shows.

Muscular Dystrophy Association (MDA) has launched a new program called MDA

Kickstart, specifically focused on developing treatments for ultra-rare neuromuscular diseases,

with a current focus on gene therapy for congenital myasthenic syndrome (CMS). This is

caused by CHAT gene deficiency, which has received both FDA Rare Pediatric Disease and

Orphan Drug designations. This initiative collaborates with UC Davis and Forge Biologics for

manufacturing support. The new MDA Kickstart Program is an in-house gene therapy program

that will partner with academic, corporate, and community groups to move these therapies

forward by creating economies of scale and improving the valuation of treatment. The MDA

Kickstart program will help ensure that patients with ultra-rare forms of neuromuscular disease

are not left behind in the era of genetic medicines and can also experience the potential benefits

of gene therapies.

One of the avenues MDA is focusing on is gene therapy which will be enriched from the

money that was raised. This is a promising therapeutic area, with such therapies approved and

many more in clinical trials. Some experts believe that gene editing holds more promise, as it

can truly alter a genetic mutation at its source and avoid some of the limitations current gene

therapy methods have. In 2023 the US Food and Drug Administration (FDA) approved the first

gene editing-based therapy for the treatment of sickle cell disease, this shows promise for

applying to MDA.

One therapy approved is Elevidys. Elevidys is a gene therapy that targets the genetic

cause of Duchenne Muscular Dystrophy (DMD). This treatment was approved by the FDA for

ambulatory and accelerated approval for non-ambulatory patients, confirming the functional

benefits of the treatment. This treatment is for children as young as 4 years old and was

recently approved to be used on adults. This therapy targets the genetic root cause of DMD and

works by introducing an engineered gene that codes for a shortened version of dystrophin,

referred to as ELEVIDYS micro-dystrophin into the muscle cells. DMD is a rare and progressive

genetic disease caused by the lack of a critical membrane–stabilizing protein called dystrophin,

which results in muscle degeneration and weakness. This disease primarily affects boys but in

rare cases, it can affect girls. The symptom occurs in early childhood between the ages of 2-3

years old.

Another treatment for DMD is Duvyzat which has also been approved by the FDA. This

treatment is an oral medication whereas Elevidys is an injection. The recent results of the Phase

3 clinical trial demonstrated a significantly smaller decline in stair climbing ability in the treated

groups as published in The Lancet. This approval of Duvyzat provides another important

treatment option for these children and young adults suffering from this disease.

This is why it is so important to continue our efforts in supporting MDA and it is exciting

to see potential improvements with our help.