REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they're developing,…
A recent study at Sanford Burnham Prebys Medical casts a ray of hope in treating multiple hereditary exostoses (MHE). MHE is a rare genetic disorder characterized by multiple noncancerous bone…
Miracle workers at Queens University are working on a revolutionary method to treat patients with Lyme disease. Lyme disease is an infectious disease that springs from a tick bite's bacteria.…
Pharmaceutical company, Pfizer Inc., announced recently that the U.S. FDA approved the first (and only) Janus kinase inhibitor for treating severe psoriatic arthritis (PsA). Called XELJANZ® and XELJANZ® XR, this…
According to a December 9th press release from Janssen Biotech, Inc, the results of a pooled analysis of patients with mantle cell lymphoma illustrated that those treated with Imbruvica (ibrutinib) had…
Pharmaceutical company and gene therapy leader, uniQure, published promising data from their current phase I/II trial testing out a gene therapy called AMT-060 in patients with serious cases of hemophilia B.…
Gene therapy has been on the horizon for years now, but before today, it was only a reality for patients with non-inherited diseases caused by genetic mutations. That just changed!…
On this season of America's Got Talent, the world met Christian Guardino. I was one of millions who heard his beautiful singing voice as he belted out his song onstage.…
Patients diagnosed with Duchenne muscular dystrophy (DMD) have always lived with a grim prognosis. Many don't make it into their 20s and face debilitating symptoms years earlier-- a dark reminder…
Robbie Edwards has a rare disease, one so rare that she's one of only 17 people in the world with it. She suffers from hereditary spastic paraplegia which represents a…
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…
The Leukemia Research medical journal published results from a study that showed efficacy when treating Acute Myeloid Leukemia (AML). Great news for the rare disease community! Onconova Therapeutics announced the publication of results…
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…
At 54 years of age, Kathleen was healthy and physically fit. She knew about her grandfather’s death at the age of 30 to a heart attack. She also knew about…
Another great breakthrough in research science! A new study found that an experimental drug combined with the traditional chemotherapy drug cisplatin successfully eliminated a rare salivary gland tumor and prevented a recurrence…
A study by the Sotiria Chest Diseases Hospital in Athens Greece revealed a surprising new link between asthma and bronchiectasis. Visit bronchiecstasisnewstoday.com to read the full article and study results.…
Neuroblastoma, though rare, is the most common type of cancer in children who are five years old or younger. This cancer targets the immature nerve cells in the nerve tissue…
Although there are treatments currently available with a proven track record against rare types of cancers, such as Waldenstrom macroglobulinemia, with any treatment, however successful, there will be a handful…
Despite a politician’s recent explanation of supply and demand by saying, “If you put the supply out there, the demand will follow,” that is not how the economic principle works.…
Life was so much simpler when we were kids. We didn’t have to worry about bills, or how what we say could accidentally offend someone else. Heck, even the stories…
NeuroVia, Inc. is a rare disease pharmaceutical company that primarily focuses on finding treatments to rare neurological diseases. Markedly, this private company is especially concerned about the effects of the…
In early July of this year, the pharmaceutical company Bristol-Myers Squibb announced that the FDA approved of their new treatment, called abatacept (Orencia), either intravenously or subcutaneously. This drug is…
Earlier this month, Vertex Pharmaceuticals released big news regarding a new development in cystic fibrosis treatment. The pharmaceutical company announced that their new, innovative technique in treating the disease had…
Ostiio, a new Philadelphia-based medical device company, has made it its mission to improve the quality of life for children that suffer from complications of craniofacial defects (in the middle of…
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