The Jack McGovern Coats' Disease Foundation, a nonprofit patient organization committed to supporting critical disease research and raising disease awareness, is offering travel grants for recipients to attend the 2020…
Sedation Versus Sleep Sleep and sedation are not synonymous terms, despite the fact that they are often used interchangeably in the hospital setting. The biggest difference? Sleep is always beneficial…
Lucy Lintott is the youngest woman in Scotland to be diagnosed with motor neurone disease (MND). As it causes progressive weakness, Lucy did not think it was possible for her…
As originally reported in Pfizer, the European Commission has just approved the first treatment option ever available to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). The medication, VYNDAQEL, taken orally once daily,…
One of the less well known forms of primary immunodeficiency is called activated PI3K delta syndrome (APDS). It is a rare disease that has only recently been identified by scientists.…
By Danielle Bradshaw from In The Cloud Copy Neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (pNFs) is a condition that many children struggle to manage due to a lack of proper…
According to a press release from Epizyme, Priority Review has been granted for tazemetostat (Tazverik), a drug used to treat relapsed or refractory follicular lymphoma (FL) for patients whose symptoms…
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According to a recent article in The Pharma Letter, the European Medicines Agency (EMA) granted PRIME (PRIority MEdicines) designation to Viralym-M based on positive results from a Phase 2 proof-of-concept…
By Jodee Redmond from In The Cloud Copy A recent study suggests that certain antibodies could be linked to specific human leukocyte antigen (HLA) genes and environmental components in scleroderma.…
By Danielle Bradshaw from In The Cloud Copy A Tufts University science team was able to identify a means to possibly reverse Friedreich’s ataxia. Friedreich’s ataxia (also known as…
By Gracie Van Brunt Hi! My name is Gracie Van Brunt, I am 25 years old and I have a rare disease. At the age of 2, I was…
By Natalie Homan from In the Cloud Copy Most people have heard of type 1 and type 2 diabetes. Many do not know, however, that there is an “intermediate” form…
According to a story from Financial Buzz, the ophthalmology company Acucela Inc. has recently announced an update on the progress of its phase 3 clinical trial. This clinical trial is…
By Natalie Homan from In The Cloud Copy Theo Thomas of Secret Harbour, Australia, may be a shy, soft-spoken 8-year-old, but he loves racing his motorcycle in his town’s Lightweight…
Poly ADP-ribose polymerase (PARP) inhibitors have played an increasing role in the treatment of ovarian cancer. They are being very heavily studied in clinical trials, both for the treatment of…
By Jodee Redmond from In The Cloud Copy Living with a chronic disease is a very stressful situation. It affects a person physically, mentally, emotionally, and spiritually. Chronic illnesses often…
According to a story from gurufocus.com, the biopharmaceutical company ADMA Biologics, Inc., has earned the 2020 BioNJ Innovator Award. This award was given as acknowledgement of the company's successful development…
According to a story from Benzinga, the biotherapeutics company CSL Behring has recently announced that its medical product PRIVIGEN ® has earned Orphan Drug designation from the US Food and…
As originally reported in CNN, a duck billed dinosaur of a time long before humanity has been identified to have suffered from the same rare disease found in children today.…
Happy Almost-Friday! This week, we're highlighting two organizations: one helps patients seeking gene therapy learn about the treatments, and the other helps families affected by a newly-recognized condition. After that,…
By Jodee Redmond from In The Cloud Copy Four United States senators have introduced a new law that, if passed, will assist children living with rare disease to receive tests…
By Danielle Bradshaw from In The Cloud Copy 13-year-old Cam Howard, a young Pittsburgh resident, recently did an interview with the Pittsburgh KDKA News’ Kristine Sorensen where he opened up…
As originally reported in BioPortfolio, a novel DNA therapy for patients with HPV 6 who experience recurrent respiratory papillomatosis (RRP) has concluded its first clinical trial with promising results. As…
One of our partner organizations, the MDS Foundation, is hosting another free MDS Patient & Family Forum in Chapel Hill, NC. When: September 26th Where: The Siena Hotel 1505 East Franklin Street…
Hypotonia Ataxia, and Delayed Development Syndrome Hypotonia, Ataxia, and Delayed Development Syndrome (HADDS) is caused by mutations in the EBF3 gene which is found on chromosome 10q26. Its discovery just…
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