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…
It's hard to believe it's already been about a month since the hemophilia A community received this exciting news! In case you missed it, there's a new drug that got…
These days there is an app for everything. There's an app for how to cook a nice meal. An app to remind you to meditate. Countless apps for finding your…
Nope, we're not joking! US SWAT teams (Special Weapons And Tactics, military weapons-equipped law enforcement teams) have been asked to step in to help with backlogged FDA applications to get experimental treatments designated as orphan…
In April, Prometic Life Sciences Inc. announced it would be submitting a Biologics License Application to the FDA for treatment of hypoplasminogenemia. The company's therapy has already been listed with Orphan…
Shortly after celebrating baby Eli’s birth, his family got the shocking news that Eli had a fatal disease called spinal muscular atrophy (SMA). Spinal muscular atrophy can take away Eli’s…
The National Health Council (NHC) and the National Organization for Rare Disorders (NORD) submitted a letter to Congressional leaders Friday - undersigned by over 120 patient advocacy organizations - urging…
An 8-year-old child in Fresno, California is receiving an FDA approved miracle drug that will possibly lengthen her life. Hayden Calafiore is the first to receive the injection as a…
If you have acromegaly, you know that available treatment options are very limited, so when Chiasma, a pharmaceutical company based in Waltham, Massachusetts, received notification that the FDA was not…
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