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…
On the surface, it seems self-evident that social collaboration can have a positive impact--especially when it comes to Duchenne muscular dystrophy (DMD). Working together with all invested participants to influence…
Parado frente a una multitud de gente hablando sobre su HFHo puede ser intimidante. Imagina que esa multitud fuese un panel de doctores y científicos que son parte de la…
In the rare disease world, nothing causes rippling waves of excitement quite like a new drug announcement. So prepare for a tsunami, because that’s exactly what you’re getting… sort of. Spring…
Recently, the FDA gave "breakthrough therapy" status to a potential new drug that may help prevent HAE attacks. But how does that affect you, the patient? What does "breakthrough therapy" really mean?…
Earlier this year, the FDA took bold steps, launching them closer to efficient rare disease drug development. And now, the potential improvement for rare disease patients could be huge. Why?…
Standing in front of a crowd and talking about your HoFH can be intimidating. Imagine if that crowd was a panel of doctors and scientists with the US Food and…
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