Barth Syndrome News and Advocacy Updates

Barth Syndrome Treatment and Advocacy Updates

“Hands down,” Tyler G. said. “The potential of this drug — this isn’t just a ‘give you some energy’ type drug. This is a ‘give you a new life’ type drug.”

STEALTH BIOTHERAPEUTICS' BARTH SYNDROME NDA RECEIVES PRIORITY REVIEW DESIGNATION
FDA Accepts new drug application from Stealth BioTherapeutics
FDA’s acceptance of Stealth NDA could be sign of improved orphan regulation
Stealth’s Elamipretide Gets US FDA Review, But Same Questions Linger
FDA agrees to review a rare disease drug that its developer was about to give up on
Stealth Bio gets FDA adcomm for previously rejected ultra-rare disease drug
MitoAction Advocates for Barth Syndrome Community in FDA Listening Session
Current Pathways For Rare Disease Drugs Are Not Optimal, US FDA's Califf Says
Companies Reveal Hurdles In Providing Drugs Via Expanded Access Programs
20+ Barth Syndrome Advocates Urged Congress to Hold FDA Accountable to Conduct Fair & Appropriate Review of Potential Treatment
WPBF News Covers Barth Syndrome story on Rare Disease Day
NEXT Report Highlights Need for FDA to exercise regulatory flexibility for Barth syndrome
Families and Advocates Rally in Washington, DC Urging FDA to Conduct Fair & Appropriate Review of Potential Treatment for Barth Syndrome
Bipartisan Letter to FDA on Ultra-Rare Diseases & Barth Syndrome
Mike Braun urges FDA to re-evaluate their approach towards ultra-rare diseases
Rare Diseases Need Treatments Too
Jamie Dubuque's Barth Syndrome Story
Patient Group Pushes FDA to Take Action on Drug
The Barth Syndrome Foundation Delivers Petition to FDA Advocating for a Fair...
SEE PETITION TO FDA HERE
Family of Arizona toddler with rare disorder fears access to life-saving therapy could end
‘We’ve exhausted all avenues’: A small biotech may give up on its ultra-rare disease drug over frustration with FDA
Rep. Tonko Expresses Concern to FDA on Inconsistent Reviews of Rare and Ultra-rare Diseases
Expanded Access: Compassion or Cop-out?
Impasse At US FDA Could Mean Stealth Abandons Barth Syndrome Treatment
Walker's Story
Jacob’s Story
Declan’s Story
Unlocking Hope: Access to Therapies for People with Rare, Progressive, and Serious Diseases
Barth Syndrome Foundation Overview
Temporal evolution of the heart failure phenotype in Barth syndrome and treatment with elamipretide
Elamipretide Prevents the Natural History Progression of Cardiomyopathy-Related Disease Symptomatology in Patients with Barth Syndrome: A Tazpower Analysis
Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome
A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism
Promising Pathway Act Would Fill Gap For Rare Disease Treatments That Don’t Fit Accelerated Approval, Advocates Say
The Promising Pathway Act is a cry for help
Dr. Hillary Vernon Speaks at a Congressional Press Conference
Emily Milligan, Executive Director at Barth Syndrome Foundation, Speaks at Congressional Press Conference
The FDA’s See No Data Approach
A Plea to the FDA to Give an Ultra-Rare Disease Drug a Fair Hearing
Mississippi’s heartbeat: A mother, her son, and their family’s battle with Barth syndrome
Community Hall lights up to observe Rare Disease Day
Milosh Kalapasev, Heart Transplant Recipient
No magic bullet’: For drugmakers and the FDA, clinical trials on ultra-rare diseases pose thorny challenges
As Needham biotech struggles with FDA, patients with an ultra-rare disease are waiting
Meet Deacon - Moments that Matter
Meet Abe - This is His Story
Parents grateful to community as baby Wally happy at home
Tiny biotech says its troubles make case for ultra-rare FDA pathway
NEW FRONTIER (FAMILY SESSIONS)
Meet Connor - A Kid with a Deadly and Invisible Disease

FDA Review Timeline

Their Words

My friend’s grandson almost died from Barth’s. He wasn’t even a year old, yet. This medication has helped to heal his heart & has finally allowed him to leave the hospital to lead a normal life.

Tesimonial-13

donator

Everyone deserves treatment options despite being rare.

Tesimonial-12

donator

I’ve watched my son struggle for 23 years. He’s beaten all odds against this deadly disease. The only time in his life he’s had a reprieve is when he was on this medication.

Tesimonial-11

donator

Access to treatment for someone with a severe disease should not be limited by how many other people have the same disease.

Tesimonial-10

donator

People with ultra-rare diseases, like Barth syndrome, need a fair chance at life-changing therapies!

Tesimonial-9

donator

My son and those suffering the same have the right to a chance of a healthy and normal life. To the world he may only be one person, but to me he’s my world!

Tesimonial-8

donator

They are sailing through life, going to school and working. They are keeping up with their peers and taking their new level of health and energy for granted. My two sons are 25% of the participants in the Elamipretide study/trial. This gift of modern medicine should be available to every affected individual who can benefit from this new medication.

Tesimonial-7

donator

If someone were to ask me today ‘Do you have Barth syndrome? I would say no, I HAD Barth syndrome.’ I am thankful for this new quality of life. I look forward to seeing how much it can help others.

Tesimonial-6

donator

Now that I’ve been on the open label extension for nearly 4 years now, I’m just happier and more pleasant to be around. I’m enjoying life more. I enjoy exercising for an hour every other day. I am able to walk one mile without taking a break – that’s four times longer than before.

Tesimonial-5

donator

As a patient that is in the TAZPOWER study my daily life is so different than just a few years ago.

Tesimonial-4

donator

There’s been a significant change of good days… Usually it was like 2 to 3 days per week. Now it’s about 4 to 5.

Tesimonial-3

donator

I feel a lot stronger. Like I’m able to do more things like than I was before the shots… Like working out or like getting to class.

Tesimonial-2

donator

He is able to walk longer distances, run a little bit more, play a little longer with his friends, and generally, has more energy.

Tesimonial-1

donator

Learn
More

Learn More

newsletter

Stay Connected

Share your email address to stay updated on ways you can support.

Barth Syndrome News and Advocacy Updates

Barth Syndrome Treatment and Advocacy Updates

FDA Review Timeline

Their Words

Tesimonial-13

Tesimonial-12

Tesimonial-11

Tesimonial-10

Tesimonial-9

Tesimonial-8

Tesimonial-7

Tesimonial-6

Tesimonial-5

Tesimonial-4

Tesimonial-3

Tesimonial-2

Tesimonial-1

Learn
More

Stay Connected

Join Us in asking for a fair, equitable and appropriate review of elamipretide in Barth syndrome by the FDA. Our voice matters! Join us

LEARN MORE

Take Action

Sign up for advocacy updates

You are now being redirected to the Barth Syndrome Foundation’s Petition on Change.org

You are now being redirected to the Barth Syndrome Foundation’s Petition on Change.org