1

March 2014
June 2014
Its not uncommon for youngsters with neuromuscular
diseases to feel isolated, as if no one understands their
physical and emotonal struggles. Partcipaton in physical
actvites is ofen limited because of the special adaptatons
required. Each year, local children, ages 6-17, atend a free
weeklong accessible summer camp where they get to be
just kids, in a supportve environment with other kids
who have similar disabilites. Actvites are geared to camp-
ers abilites, and each camper is assigned a trained, full-tme
volunteer assistant.
This weeklong summer camp is also an opportunity for
parents to get a welcomed break from their role as full-tme
caregivers and they are refreshed and ready to resume their
responsibilites afer camp.
MUSCLES
MATTER

MUSCULAR DYSTROPHY ASSOCIATION
508-898-3375
boston@mdausa.org
33 Lyman Street, Suite 205, Westborough, MA 01581


Index

1. Summer Camp/Staf
2. Highlights
3. Additonal Highlights
4. Fill The Boot Drives
5. Research Updates
6. Highlighted Upcoming Events
7. Upcoming Events
Vanessa Malftano
Area Director
Kate Tighe
Area Director
Britany DeFrancesco
Fundraising Coordinator
Brad Gosselin
Fundraising Coordinator
Will Southall
Fundraising Coordinator
Molly McDermot
Health Care Coordinator
Lindsay Johnson
Endurance Manager
Lucia Doucete
Administratve Assistant
Amy LHeureux
Administratve Assistant
Kate Macomber
Administratve Assistant
Massachusets Chapter Staf
David Spellman
Director of Health Care Services
2

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

Evening of Hope
Held - May 29th, 2014 at Doubletree Hotel by Hilton, Westborough, MA
3
24-Hour Muscle Walk Challenge







Held June 9th at Bass Rocks Country Club

to all who supported
and took part in
this event
which raised




T
H
A
N
K

Y
O
U


to
e
v
e
ry
o
n
e

w
h
o
s
u
p
p
o
rte
d

M
D
A
in
th
is

s
u
c
c
e
s
s
fu
l w
a
lk
!!
A special thanks to Deb LaFrance
for being the driving force
behind this event!
O
v
e
r $
2
5
,0
0
0
R
a
ise
d
!
A
n
In
cre
a
se
O
f
$
7
,0
0
0
F
ro
m
L
a
st Y
e
a
r!!
4

















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










5
Stay connected to MDAs 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
conditonal 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 patents with Duchenne muscular dystrophy (DMD) who can be helped by skipping exon
(secton) 51 of the dystrophin gene, says it is actvely 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 fow
to exercising muscles is defcient and that treatment with either tadalafl (Cialis) or sildenafl
(Viagra) normalizes this blood fow, at least in the short term (afer one dose of either drug)
How Do Neuromuscular Disorders Affect People's Lives?
Researchers at the University of Michigan are interested in collectng patents impressions of how
genetc neuromuscular disorders afect their lives, partcularly with respect to pain, depression, fa-
tgue, sleep abnormalites and cognitve 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 functonal 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 efcacy of gene transfer therapy in infants with type 1
spinal muscular atrophy (SMA) who are 9 months old or younger has opened at Natonwide
Childrens Hospital in Columbus, Ohio, under the directon of neurologist Jerry Mendell.
Tirasemtiv May Slow Respiratory Decline in ALS
The experimental drug trasemtv, in development by South San Francisco biopharmaceutcal compa-
ny Cyokinetcs as a potental treatment for amyotrophic lateral sclerosis (ALS), slowed the decline in
at least one measure of respiratory functon in a recent, large-scale study known as BENEFIT-ALS
(Blinded Evaluaton of Neuromuscular Efects and Functonal Improvement with Tirasemtv in ALS)...
Shape of Genetic Material Matters in C9ORF72-Related ALS
A mutaton in a gene located on chromosome 9 and known as C9ORF72 is the most common genetc
cause of amyotrophic lateral sclerosis (ALS), but exactly how it causes this disease has been unclear
(Please right click on ttle for each artcle and click Open Hyperlink to view entre artcle)
6
7
June 25
Springfield Lock-Up
Samuels
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.Massachusets twiter.com/MDA_Mass

MDA/ALS Support Group
Where: 33 Lyman Street, Suite 205, Westborough, MA 01581
When: TBD
For more informaton please contact Dave Spellman at (508) 898-3375 or dspellman@mdausa.org