Asklepios BioPharmaceutical, Inc. - AskBio

04/05/2022

Asklepios BioPharmaceutical, Inc. - AskBio is supporting Parkinson’s Awareness Month with some important facts to help raise awareness about the disease.

Parkinson’s disease is a progressive, incurable neurological disorder associated with a loss of dopamine-generating and other brain cells that results in a complex array of symptoms. It is primarily associated with progressive loss of motor control, but there are many troublesome non-motor symptoms.

Learn more facts and help raise awareness by supporting the efforts of the Michael J. Fox Foundation: https://www.michaeljfox.org/

03/16/2022

At this week’s MDA Clinical & Scientific Conference, investigators from Duke University School of Medicine and Duke Clinical Research Institute presented data from a Phase 1 Study of Gene Therapy in Late-onset Pompe Disease.

In the study, all subjects treated with gene therapy with an adeno-associated virus serotype 8 (AAV8) vector (AAV8-LSPhGAA) demonstrated sustained serum GAA levels above baseline trough activity two weeks following the preceding enzyme replacement therapy (ERT) dose which confirmed bioactivity.

These initial data support the safety and bioactivity of AAV8-LSPhGAA and justify continued clinical development of AAV8-LSPhGAA therapy in Pompe disease patients.

To learn more about this trial, please visit: https://bit.ly/3wcIteF

03/15/2022

March is Multiple System Atrophy (MSA) Awareness Month, and Asklepios BioPharmaceutical, Inc. - AskBio joins with the Defeat MSA Alliance to help raise awareness of this rare, rapidly progressing and devastating neurodegenerative disorder that affects the brain.

Learn more about MSA, the MSA Alliance and its efforts to worldwide: https://bit.ly/3Ie7s3L

02/28/2022

Today, on Rare Disease Day, we are showing our support for the more than 300 million people around the world living with rare diseases. This includes those living with Pompe disease, Limb Girdle Muscular Dystrophy, Multiple System Atrophy and Huntington’s disease.

We hear you, and we support you.

09/15/2021

Today marks International Myotonic Dystrophy Awareness Day and AskBio is proud to support the Myotonic Dystrophy Foundation in its efforts to raise awareness of this devastating genetic disease.

Myotonic dystrophy, or DM, is a rare, multi-systemic, inherited disease that affects an estimated 1 in 2,100 people, over 3.6 million individuals across the world. Help us improve awareness on behalf of Learn more and support the cause https://bit.ly/3xIIFk1

04/15/2021

As we mark , AskBio honors all of the patients and families living with this devastating disease, including Dakota. Dakota is one of the first patients to survive with infantile Pompe thanks to screening and treatment. Learn more about Dakota’s story here: https://bit.ly/3mTrgQL

04/14/2021

Join AskBio as we celebrate International Pompe Day on April 15th. TOGETHER WE ARE STRONG!

02/17/2021

Read about the need for new treatment paradigms for ultra-rare diseases. Columbus Children's Foundation Chairman/CSO Jude Samulski, Sanath Ramesh, founder & CEO of OpenTreatments Foundation, and Max Bronstein, health policy expert, highlight a path so that while these kids are rare, they are not forgotten! https://www.statnews.com/2021/02/17/new-drug-development-paradigms-ultra-rare-diseases

The technology to find treatments for rare and ultra-rare diseases exists today. The world needs a drug development paradigm for them all.

02/03/2021

Gearing up for next week? We invite you to stop by our booth to learn more about and our in disease. Register at https://worldsymposia.org.

01/07/2021

Wonderful news today from Pfizer announcing the start of Phase 3 trials for patients with muscular dystrophy. We're honored to have played an early role in the development of the treatment and applaud this important step for patients. http://bit.ly/39b4Wf1

Pfizer Inc. (NYSE: PFE) today announced that the first participant has been dosed in the Phase 3 CIFFREO study, which will evaluate the efficacy and safety of investigational gene therapy candidate PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The CIFFREO trial is expected to enroll 99...

11/30/2020

While holds great promise for people with a , the pursuit of these one-time treatments may not be economically viable to some commercial drug developers. In the case of ultra-rare conditions, the patient populations may be so small that it may not be possible for companies to recoup their investments, let alone make a profit. The nonprofit Columbus Children's Foundation is providing funding and scientific resources to accelerate access to gene therapies for children affected by these disorders. Global Genes spoke to AskBio's Jude Samulski, a gene therapy innovator, about the Foundation, the range of capabilities it can bring together, and the potential to scale what it does to reach more in need of treatments. https://bit.ly/2VgolEO

While gene therapies provide great promise for people with rare, genetic diseases, the pursuit of these one-time treatments may not seem economically viable to commercial developers. In the case of ultra-rare conditions, the patient populations may be so small that it may not be possible for compani...