Currently, there are an estimated 30 million people within the European Union (EU) who have a rare disease. The “Orphan Medicines” (or Orphan Drug) program was designed to facilitate…
David Guy, parent of Kaila who was diagnosed with acid sphingomyelinase deficiency (ASMD), recounts his initial shock when he was told of Kaila's rare disease. David and his wife…
When we ‘do the math’ it adds up to the novel AMT-130 gene therapy receiving positive results from uniQure’s clinical trial investigating a small group (N=4) of individuals who were…
Dr. Jung-Mo Ahn, associate professor at Dallas University in Texas, created a molecule that kills a broad range of treatment-resistant cancers. The newly targeted group includes triple-negative breast cancer.…
Within the first few months after Reuben Jayce-Mills was born, he seemed to be progressing fairly normally. But when Reuben turned nine months old, his parents noticed some concerning…
Actor Max Adler is known for a variety of roles in films and TV shows such as Glee, Saugatuck Cures, and Switched at Birth. But did you know that…
In June 2022, the FDA approved Amvuttra, a prescription medicine designed to treat polyneuropathy (nerve damage) due to hereditary transthyretin amyloidosis (hATTR amyloidosis). This approval was huge. Typically, managing…
According to a story from Buzzfeed News, Comedian Sandy Honig, best known for writing and starring in Three Busy Debras, was recently diagnosed with gastroparesis, a rare disease that impacts…
Business Wire published an August 10th news release announcing that Cure Rare Disease, a non-profit based in Boston, Massachusetts received FDA approval to administer CRD-TMH-001 (CRD), its first-ever therapeutic.…
According to a story from Benzinga, the pharmaceutical company Inceptua Group has recently announced that the medicine paclitaxel micellar (marketed as Apealea) is now available in Germany. This medication is…
According to a story from Market Screener, the biotech company UNITY Biotechnology, Inc., has recently announced the release of data from 12 weeks and 18 weeks of its Phase 2…
According to a story from Upstate Medical University, the college's ALS Treatment and Research Center has been a certified center of excellence for research and clinical care that has helped…
Last year, Mandy Moore, 38, celebrated as she brought her son Gus into the world. Known for her roles in This Is Us, A Walk to Remember, and Saved (among many others), Mandy…
According to a story from Kalimpong News, over 300 children with rare diseases in India are still waiting for the release of pledged funds from the Union Health Ministry so…
According to a story from Market Screener, the clinical stage biopharmaceutical company Kringle Pharma, Inc., has recently announced an update on the progress of its phase 2 clinical trial. The…
Karyopharm Therapeutics, a pharmaceutical company focusing on novel cancer therapies, has issued a statement via PR Newswire that the FDA has granted regulatory designations to eltanexor, Karyopharm’s Selective Inhibitor…
According to a story from Medical Xpress, a medicine used to treat high blood pressure and enlarged prostate could be a potential therapy for amyotrophic lateral sclerosis (ALS), a rare…
On August 10, 2022, KTVU anchor Leslie Griffith passed away in Lake Chapala, Mexico, where she had been living for the past six years. Leslie was 66 years old…
Near the start of August 2022, biotechnology company Inmagene Biopharmaceuticals ("Inmagene") and biopharmaceutical company HUTCHMED shared via news release that the first participant had been dosed in a Phase…
According to a story from Ankylosing Spondylitis News, Health Canada has recently approved upadacitinib (marketed as Rinvoq) as a treatment for people living with ankylosing spondylitis. The medicine is specifically…
For those living with sickle cell disease (SCD), treatment and management can sometimes be difficult. Currently, there is no cure for SCD. Patients may manage their condition using antibiotics,…
According to a story from express.co.uk, the COVID-19 pandemic was a shock to the world when it first struck over two years ago, and while we have a better understanding…
According to a recent press release from Globe Newswire, Albireo Pharma announced that Lancet Gastroenterology published results of the PEDFIC 1 trial evaluating Bylvay® in the treatment of progressive…
According to a story from Charcot-Marie-Tooth News, the Global CMT Research Convention is set to take place in Cambridge, Massachusetts on September 16-17, 2022. Organized by the CMT Research Foundation,…
Currently, there are an estimated 30 million people in the European Union (EU) who are living with a rare disease. Drug developers are incentivized to create therapies for those…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
