According to a press release from the biopharmaceutical company Sanofi, the company's investigational treatment avalglucosidase alfa will have its Biologics License Application (BLA) evaluated by the US Food and Drug…
According to a press release from the biopharmaceutical company Sanofi, the Biologics License Application (BLA) for its drug avalglucosidase alfa has been accepted by the US Food and Drug Administration…
Patient registries are a great way for patients to get involved in shaping future research around their conditions. Many times, getting involved in a rare disease (or rare disorder)…
AVROBIO AVROBIO is based out of Cambridge, Massachusetts. They specialize in single-dose gene therapy as a cure for rare genetic conditions such as cystinosis, Gaucher disease, and Fabry disease. Most…
Every day, medical researchers look to learn more about rare diseases, symptoms, and treatment. Recently, the Muscular Dystrophy Association (MDA) connected with Dr. Priya Sunil Kishnani, MD, MBBS to discuss what…
According to Batten Disease News, Invitae and Axovant Gene Therapies partnered up to offer free genetic testing in North America. This will focus on children who are thought to…
So you're looking to get pregnant or curious about some part of it. Well, you've come to the right place! If you're looking to have a baby, but you also…
April 15th, 2020 will be the 7th annual International Pompe Day, a day of recognition and fostering awareness around Pompe disease organized by the International Pompe Association. Like many rare…
Sanofi There are an estimated 350 million rare disease patients across the world. Many conditions still don't have treatments. However, there are scientists dedicating their lives solely to this endeavor.…
By Danielle Bradshaw from In The Cloud Copy Pompe disease is the lack of a specific protein within the body that helps break down glycogen - a complex sugar that…
According to a story from Sixth Tone, life for the Lu family of Yuncheng, China, has never been easy. Life in the region has slowed thanks to the coronavirus, but…
AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina. This company is dedicated to the research of gene therapies as potential therapeutic options…
According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…
According to a story from tradeshownews.vporoom.com, the Muscular Dystrophy Association (MDA) has recently announced the official approval of its Patient-Focused Drug Development (PFDD) meeting by the US Food and Drug…
By Jodee Redmond from In the Cloud Copy The results of a recent study published in Pompe Disease News suggest that a molecular “tag” used to improve the distribution of…
Actus Therapeutics, a privately held portfolio company of AskBio, has recently announced through PRWeb, the initiation of patient dosing in a clinical trial testing its investigational gene therapy ACTUS-101. About Pompe…
According to a story from birminghammail.co.uk, eight month old Archie was diagnosed with a serious rare disorder called Pompe disease. With his life in danger, the infant faces 220 mile…
According to a story from healthbeat.spectrumhealth.org, Tasha Veinbergs was 13 years old when she was first diagnosed with Pompe disease. She was visiting the doctor to treat a small cyst…
According to a story from Pompe Disease News, a recent study has concluded the polymorphisms of the ACE gene (which are genetic variations that occur naturally) do not have an…
According to a story from Pompe Disease News, a recent study has found that delays in diagnosis are closely connected to whether someone displaying symptoms of Pompe disease is referred…
Wearable monitoring technology that tracks a persons movement, heartbeat, and bodily functions has become popular across society because it gives us a window into our bodies and to watch the…
According to a publication from Fabry Disease News, Amicus Therapeutics has as many as 14 investigational drugs currently in development for the treatment of rare diseases including Fabry, Pompe, and…
Extended Collaboration Amicus Therapeutics has just announced that they are expanding their collaboration with the University of Pennsylvania's Perelman School of Medicine for the next five years. This collaboration is…
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In about five years India’s population will exceed that of China which is currently ranked number one according to population. Contrast these numbers with the fact that only ten percent…
According to a story from Healthworld, the government of Kerala, an Indian state located on the southwestern coast, has announced plans to take action in order to treat patients with…
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