Not so rare

Meet Tim. Tim is Tom’s little brother. From birth he had some health complications, but quickly recovered. However, like Tom he had trouble with his speech at the age of 2. The explanation? “He’s just like Tom.” Read more about brothers Tim and Tom on our blog and follow the patient journey and lengthy diagnosis for a child living with a rare disease.

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Andy JobsonNot so rare

Not So Rare

Tom and Tim finally reach a diagnosis after finding genetic faults known as AGU. The doctor couldn’t tell them much more, so they were sent to a rare disease specialist for further consultations. For Tom and Tim, and their families, it’s been a long process to reach a clear diagnosis. Sadly, like many other rare diseases, there is no therapy or treatment available. Rare diseases affect more than 350 million people worldwide. We need to support people like Tom and Tim to raise awareness of the long and stressful journey through to diagnosis, to understand the reality of living with a rare condition and to support those families affected by a rare disease.

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Andy JobsonNot So Rare
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Not so rare

Today is #RareDiseaseDay. Did you know a patient will end up visiting several doctors before getting an accurate diagnosis? Let’s work together to heighten awareness #notsoRARE Engel PA, Bagal S, Broback M, Boice N. Physician and patient perceptions regarding physician training in rare diseases: The need for stronger educational initiatives for physicians. Journal of Rare Disorders, 2013; 1(2): 1-15.

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Andy JobsonNot so rare
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Not so rare

Research into #rarediseases can save and change lives. On #RareDiseaseDay let’s have a common cause to help increase awareness. Engel PA, Bagal S, Broback M, Boice N. Physician and patient perceptions regarding physician training in rare diseases: The need for stronger educational initiatives for physicians. Journal of Rare Disorders, 2013; 1(2): 1-15

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Andy JobsonNot so rare

Upcoming Rare Disease Blog posts

In support of #RareDiseaseDay we are launching a series of blog posts to illustrate the length of time it takes for a child living with a rare disease to receive a diagnosis. The process will be reviewed, from the parent’s perspective looking at the initial symptoms and beyond. Follow the link to find out about Tom. https://onyx-notsorare.tumblr.com/ Stay tuned over the next couple of weeks to find out what happens. #notsoRARE Engel PA, Bagal S, Broback M, Boice N. Physician and patient perceptions regarding physician training in rare diseases: The need for stronger educational initiatives for physicians.

Journal of Rare Disorders, 2013; 1(2): 1-15.

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Andy JobsonUpcoming Rare Disease Blog posts
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Amazing scientific sight…! The teams from ONYX and Only Oncology take a few moments on a recent business trip to admire the Atomium as it gleams against the bright blue Brussels sky!

Picture + Amazing scientific sight! Thanks to ONYX and Only Oncology for sharing this fantastic pic taken on a recent business trip.

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Prime GlobalAmazing scientific sight…! The teams from ONYX and Only Oncology take a few moments on a recent business trip to admire the Atomium as it gleams against the bright blue Brussels sky!

Eteplirsen approved in US for Duchenne muscular dystrophy

The first drug to treat the underlying cause of Duchenne muscular dystrophy, Exondys (eteplirsen) developed by Sarepta Therapeutics, has secured marketing approval from the U.S. Food and Drug Administration. Duchenne muscular dystrophy is a severe type of muscular dystrophy which generally begins around age four in boys and progresses rapidly.

www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm

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Prime GlobalEteplirsen approved in US for Duchenne muscular dystrophy

StrimvelisTM receives European marketing authorisation to treat very rare disease ADA-SCID

Strimvelis, (autologous CD34+ cells transduced to express ADA) the first ex vivo stem cell gene therapy has been approved to treat patients with ADA-SCID (severe combined immunodeficiency due to adenosine deaminase deficiency). Children born with ADA-SCID do not have a heathy, fully functioning immune system and are unable to successfully combat everyday infections.

www.gsk.com › en-gb › Media › Press releases

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Prime GlobalStrimvelisTM receives European marketing authorisation to treat very rare disease ADA-SCID
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Onyx participates in World Orphan Drugs Congress in Washington

Following a successful presentation at the Cell Therapy Manufacturing and Gene Therapy Congress in Brussels, Onyx has again had a strong presence at the prestigious World Orphan Drugs Congress in Washington. This emphasises Onyx strong and continuing commitment to the areas of orphan drugs, rare and ultra-rare diseases and regenerative medicine including cell and gene therapies.

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Prime GlobalOnyx participates in World Orphan Drugs Congress in Washington