FDA Will Review Translarna for DMD
PTC Therapeutics announced that the FDA will review its investigational drug ataluren (brand name Translarna) for the treatment of some forms of DMD.
Strongly
An expression of the live unlimited spirit, stories and voices of the MDA community
Keeping Up the Momentum
For this latest round of awards, MDA reviewed 187 applications and had sufficient funds to approve funding for approximately 15 percent of them — 29 grants with a total funding commitment of more than $7 million.
Team Momentum athlete takes on Boston in honor of family members with CMT
Colin Batty will be running with MDA Team Momentum in the Boston Marathon to raise funds in honor of family members who live with CMT.
Seeing the World Thanks to MDA Shamrocks: Meet Gabrielle, the Traveling Shamrockstar
Fourteen-year-old Kentucky native Gabrielle’s favorite subject is geography, which is appropriate when you have an insatiable desire to travel. Gabrielle, who has spinal muscular atrophy (SMA) Type 2, has already seen a great deal of the world, including Europe, the Caribbean and the western United States. This year, Gabrielle is featured as a “Shamrockstar” on platinum MDA Shamrocks at retailers across the country, a country that Gabrielle has seen extensively in her travels with her family.
Keeping Up the Momentum
Following the approval of three drugs in six months, new MDA grants push for more progress By Grace Pavlath, Ph.D., MDA Senior Vice President & Scientific Program Director What are the effects of steroids on muscle repair in patients with Duchenne muscular dystrophy (DMD)? How can gene therapy impact people with Charcot-Marie-Tooth disease (CMT)? Will . . .
MDA Outlines Principles of Coverage for ACA Replacement with Coalition of Patient Groups
As policy makers are considering various options to modify or replace the Affordable Care Act (ACA), MDA is working to ensure that individuals and families affected by chronic, serious and life-threatening diseases maintain health care access and coverage.
Denise’s Dodgers: Dodgeball Tournament Targets ALS, Raises Funds in Honor of Beloved Illinois Woman
Inspired by a beloved community member with ALS, this Illinois dodgeball tournament is good fun for a good cause.
MDA Recommends SMA Be Added to Newborn Screening Nationwide
Many families whose children have spinal muscular atrophy (SMA) endure a long and difficult diagnostic odyssey. This may soon change, however, based on recommendations MDA recently made with a coalition of partners that SMA should be added to the list of conditions newborns are screened for at birth in the United States.
“There’s No Better Feeling in the World than Having Your Child Cared for by Someone who Loves Him Like You Do”
The Labbadia family never wanted to need an MDA Care Center, but when son Brendan was diagnosed with Duchenne muscular dystrophy, they found that their team at Nemours Children’s Hospital in Orlando, Fla., including pediatric neurologist Dr. Richard Finkel, was the one bright spot of an otherwise devastating journey.
A Strong Future: Shamrockstar Maya Benefits from New Treatments for SMA
Over the past 35 years, the MDA Shamrocks program has raised more than $300 million to help save and improve the lives of families living with muscular dystrophy. Get to know Maya, who graces the 2017 green MDA Shamrock.
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