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March 2014 June 2014

It’s not uncommon for youngsters with neuromuscular diseases to feel isolated, as if no one understands their physical and emotional struggles. Participation in physical activities is often limited because of the special adaptations required. Each year, local children, ages 6-17, attend a free weeklong accessible summer camp where they get to be “just kids,” in a supportive environment with other kids who have similar disabilities. Activities are geared to camp-ers’ abilities, and each camper is assigned a trained, full-time volunteer assistant.

This weeklong summer camp is also an opportunity for parents to get a welcomed break from their role as full-time caregivers and they are refreshed and ready to resume their responsibilities after camp.

MUSCLES MATTER

MUSCULAR DYSTROPHY ASSOCIATION

508-898-3375 boston@mdausa.org

33 Lyman Street, Suite 205, Westborough, MA 01581

Index

1. Summer Camp/Staff

2. Highlights

3. Additional Highlights

4. Fill The Boot Drives

5. Research Updates

6. Highlighted Upcoming Events

7. Upcoming Events

Vanessa Malfitano

Area Director

Kate Tighe

Area Director

Brittany DeFrancesco

Fundraising Coordinator

Brad Gosselin

Fundraising Coordinator

Will Southall

Fundraising Coordinator

Molly McDermott

Health Care Coordinator

Lindsay Johnson

Endurance Manager

Lucia Doucette

Administrative Assistant

Amy L’Heureux

Administrative Assistant

Katie Macomber

Administrative Assistant

Massachusetts Chapter Staff

David Spellman Director of Health Care Services

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MORTON H. SIGEL MEMORIAL AWARD

PRESENTED TO: ERA Key Realty Services

JAMES E. WALLACE JR., EVENING OF HOPE SPIRIT AWARD

PRESENTED TO: Matthew R. Bruce (In memorium)

MDA LOU GEHRIG HUMANITARIAN AWARD

PRESENTED TO: Merit Cudkowitz, MD

MDA’s ALS Evening of Hope

Held - May 29th, 2014 at Doubletree Hotel by Hilton, Westborough, MA

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24-Hour Muscle Walk Challenge

Held June 9th at Bass Rocks Country Club

THANK YOU!

to all who supported

and took part in

this event

which raised

Additional Highlights

THANK YOU

to everyone

who supported

MDA in this

successful walk!!

A special thanks to Deb LaFrance

for being the driving force

behind this event!

Over $25,000 Raised!

An Increase Of

$7,000 From Last Year!!

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Waltham Fire Local 866 on raising $7,500 on their Fill The Boot on June 16th!!

Congratulations!

Watch for upcoming Fill the Boot drives.

Check us out on Twitter and Facebook for

more information!!

Registration: 8am Tee-Off: 9am Cost: $125 per golfer Includes 18-Holes, Golf Cart, Collectible CFD gift, Lunch and Prizes.

This event is open to all CFD Local 30 members, Family and Friends.

*Hole Sponsorship is also available*

Upcoming Fill the Boot

June 28th - Marblehead Fire

June 28th - Tewksbury Fire

July 4th - Dracut Fire

July 19th - Peabody Fire

July 19th - Hanscom Airforce Base

July 26th - Newburyport Fire

July 27th - Newburyport Fire

Fire Department Events

June 29th - Belmont Fire Golf Tournament

July 9th - Cambridge Fire Golf

July 12th - Malden Fire Bowl-a-thon

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Stay connected to MDA’s worldwide research team, and get the latest research news online at www.mda.org/research!

DMD: Ataluren May Get Conditional Approval in Europe

The experimental Duchenne muscular dystrophy (DMD) drug ataluren (which has been given the

brand name Translarna) has received encouragement from the European Medicines Agency (EMA) for

conditional approval in European Union countries…

DMD: Prosensa Pursuing Path Forward for Drisapersen Dutch biotechnology company Prosensa, developer of the experimental exon-skipping drug drisaper-

sen for patients with Duchenne muscular dystrophy (DMD) who can be helped by skipping exon

(section) 51 of the dystrophin gene, says it is actively working to determine a path forward for this

drug…

Tadalafil, Sildenafil Increase Blood Flow to DMD-Affected Muscles A study conducted in 10 boys with Duchenne muscular dystrophy (DMD) has found that blood flow

to exercising muscles is deficient and that treatment with either tadalafil (Cialis) or sildenafil

(Viagra) normalizes this blood flow, at least in the short term (after one dose of either drug)…

How Do Neuromuscular Disorders Affect People's Lives? Researchers at the University of Michigan are interested in collecting patients’ impressions of how

genetic neuromuscular disorders affect their lives, particularly with respect to pain, depression, fa-

tigue, sleep abnormalities and cognitive problems...

SMA: ISIS-SMNRx Shows Benefit in Infants, Children Treatment with the experimental drug ISIS-SMNRx in two phase 2 studies in infants and children with

spinal muscular atrophy (SMA) has been found to result in functional improvement, encouraging its

developer to pursue phase 3 studies in 2014….

First Human SMA Gene Transfer Therapy Trial Opens A phase 1 trial to test the safety and efficacy of gene transfer therapy in infants with type 1

spinal muscular atrophy (SMA) who are 9 months old or younger has opened at Nationwide

Children’s Hospital in Columbus, Ohio, under the direction of neurologist Jerry Mendell….

Tirasemtiv May Slow Respiratory Decline in ALS The experimental drug tirasemtiv, in development by South San Francisco biopharmaceutical compa-

ny Cyokinetics as a potential treatment for amyotrophic lateral sclerosis (ALS), slowed the decline in

at least one measure of respiratory function in a recent, large-scale study known as BENEFIT-ALS

(Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS)...

Shape of Genetic Material Matters in C9ORF72-Related ALS A mutation in a gene located on chromosome 9 and known as C9ORF72 is the most common genetic

cause of amyotrophic lateral sclerosis (ALS), but exactly how it causes this disease has been unclear…

(Please right click on title for each article and click Open Hyperlink to view entire article)

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June 25

Springfield Lock-Up

Samuel’s Springfield, MA

mdalockup.org/springfieldLU

July 6-11

Summer Camp Camp Allen Bedford, NH

July 9 Cambridge Fire Golf Tournament

Wayland Country Club Wayland, MA

July 12 Malden Fire Bowl-A-Thon

Town Line Ten Pin Malden, MA

July 12-18

Summer Camp Camp Waban Sanford, ME

July 28

Acosta Golf Tournament The International

Bolton, MA

July 27 - August 1

Summer Camp Channel 3 Camp

Andover, CT

September 14

Team Momentum Half Marathon Boston, MA

boston131.com

Week of August 25

Show of Strength Preview Party Stay tuned for more details

September 15

Marlborough Fire Local 1714 Golf Tournament Marlborough, MA

(open to the public)

October 19

Halloween Witch Ride Boston Harley-Davidson

Everett, MA

mdawitchride.com

November Muscle Summit

TBA

Facebook.com/MDA.Massachusetts twitter.com/MDA_Mass

MDA/ALS Support Group

Where: 33 Lyman Street, Suite 205, Westborough, MA 01581 When: TBD

For more information please contact Dave Spellman at (508) 898-3375 or dspellman@mdausa.org