If drugs were measured by the quantity of approvals, orphan drugs would make quite a splash. Measured in more financial terms, however, they make up a much smaller portion of…
Happy Friday everyone! The leaves are beginning to change, and so are ongoing activities at the FDA. This week, we're highlighting four FDA announcements we think you should hear about.…
Happy October, everyone! We hope everyone's enjoying this new ~fall weather~ and the endless supply of pumpkin spice flavored food products. This week, we're highlighting four FDA announcements we think…
Lindsey Sutton showed signs of familial chylomicronemia syndrome as early as five weeks old. Sutton is now 28 years old. Because of her condition, she must adhere to a very…
Happy Friday, everyone! We're trying something new this week. Instead of wrapping up with an Editor's Choice article, we're highlighting four FDA announcements we think you should know. Check them…
Discussion on Right to Try legislation continues, and the federal government could soon change existing rules. Many people wonder whether or not patients should have the right to try potentially…
New medicines for Parkinson’s may be around the corner. The United States Food and Drug Administration (US FDA) recently approved one drug, and accepted application another. Both new drugs treat…
The FDA has doubled down on warnings regarding balloon angioplasty. Proposed as a treatment for multiple sclerosis, the surgical use of balloons in the jugular vein has been deemed risky…
The FDA has granted a special designation for a drug company developing a gene therapy for the treatment of leber congenital amaurosis (LCA). MeiraGTx received rare pediatric disease designation for its…
Great news for the rare disease community! Ultragenyx Pharmaceuticals reported back positive results from its phase 3 study of the treatment drug burosumab in adults with X-linked hypophosphatemia (XLH). XLH…
Lilia Zaharieva was a University of Victoria student in her early late twenties when she found herself losing half her leg function due to cystic fibrosis. Walking from class to…
A major first step for a viable treatment option! Achillion Pharmaceuticals last week reported preliminary proof-of-concept results from its ongoing Phase 2, study of a new treatment for C3 glomerulopathy…
Earlier this month, the FDA approved Zelboraf (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. The final results indicated a response rate of 54.5%. This is the first FDA-approved treatment…
Big news for those with hemophilia! On Thursday, the FDA approved Hemlibra (manufactured by Genentech), a weekly self-injection for hemophilia A patients who have developed resistance to standard medicines for preventing…
Sam and Anna Beiler have not one, but two children with a rare degenerative disease that causes blindness. As of now, there is no treatment. Last month, however, the FDA…
A spokesperson for Sarepta Therapeutics announced that their latest drug for the treatment of Duchenne muscular dystrophy is doing very well and is actually helping patients. Duchenne muscular dystrophy (DMD)…
While other kids his age run amok in the wildness of their Terrible Two's, Lincoln Woodmass remains immobile. No running, no jumping and the possibility of never walking again. You…
The FDA makes decisions that determine the lives and treatments of patients with rare diseases. Many of these people who make these decisions aren't impacted by the disease in their…
A Futurism article this week put into very human terms the ongoing question we face when it comes to rare disease treatment and Pharma: What is the cost of life?…
The FDA this week granted orphan drug designation to glepaglutide for the treatment of short bowel syndrome (SBS), developed by Zealand Pharma A/S. SBS is a rare disorder where an…
Biogen's third quarter earnings report isn't looking good these days, putting an uneasy cloud over the biotech sector as a whole. But there is a light at the end of…
There's a new treatment for X-Linked Hypophosphatemia (XLH) that may be available in the not-so-distant future. XLH occurs when the kidneys process phosphate abnormally, leading to excess phosphate loss in…
Prescription drugs have always been overly expensive but there's now hope for these prices to drop down. On Tuesday morning, the Senate Health Committee held a hearing to discuss the…
A recent report from the Food & Drug Administration reveals renewed efforts for the treatment of one of the rarest forms of non-Hodgkin's Lymphoma, Mantle Cell Lymphoma. Due to promising advancement…
It's been a tough year for the United States Congress and healthcare legislation - you might have heard, they failed to repeal and replace the Affordable Care Act nor have…
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