The long-awaited decision by the European committee CHMP was announced recently by Amylyx Pharmaceutical (the Company) based in Cambridge, Massachusetts just as anticipated. The EMA issued its negative opinion of…
People in China who are living with narcolepsy will soon have access to a new therapeutic option to help them manage cataplexy or excessive daytime sleepiness (EDS) after the…
Patient Worthy partner Chelsea's Hope is a nonprofit organization dedicated to supporting families impacted by the deadly rare illness Lafora disease, as well as amplifying research. Recently, a documentary titled Fighting…
According to a story from Vox, Americans are some of the world's most voracious meat eaters. Per capita, Americans eat an average of 60 lb. of red meat each year,…
According to a story from Market Screener, the drug company AbbVie recently announced that its investigational therapy epcoritamab has received a positive opinion from the European Union's Committee for Medicinal…
According to a story from WGN-TV Chicago, treatment for esophageal cancer can be grueling on patients, even if successful. Surgery is often a critical aspect of the process and can…
Prothena Corporation which is based in Dublin, Ireland recently announced, via Business Wire and Biospace results of the VITAL Phase 3 clinical trial showing a significant survival benefit…
Findings from the Max Delbrück Center and the Charité Universitätsmedizin Berlin have been published in the European Respiratory Journal stating that a triple combination therapy can improve CF symptoms…
If your child was sick, you’d do everything that you could to save them. Hayley Moore is no different. When her daughter Klarissa was born, Moore felt such a…
According to a report in the morningstar.com, the FDA has approved AstraZeneca/Sanofi shot that protects infants and toddlers against RSV. The 2022/2023 winter saw an RSV increase, especially among…
According to a story from European Pharmaceutical Review, the European Commission has recently approved a label extension of ORKAMBI (lumacaftor/ivacaftor), a drug used to treat cystic fibrosis. Under the extension,…
According to a July 11 article from Maaisha Osman, commercial-stage biotechnology company ADC Therapeutics has voluntarily halted enrollment for the Phase 2 LOTIS-9 study evaluating Zynlonta (loncastuximab tesirine-lpyl) and…
On the field at the University of Tennessee, defensive back (DB) Trevon Flowers was known for his intense gameplay, strong athletic ability, and defensive nature. He appeared in 46…
According to an a press release published on Business Wire, Arrowhead Pharmaceuticals Inc. recently presented updated phase 2 data at the 2023 European Association for the Study of the Liver…
In March 2023, the European Commission (EC) approved Pombiliti (cipaglucosidase alfa) for the treatment of adult patients living with late-onset Pompe disease. Just three months later in June, another…
American consumers have access to the most advanced pharmaceutical systems in the world. Recent medical and technical advances seem to support that reputation. According to Medical Xpress, a Swiss…
According to a story from International Business Times, a French couple encountered an unpleasant skin condition as a result of a pest infestation in their home. Their case was reported…
The FDA has approved Rystiggo, a developed by UCB in Brussels, to treat two types of myasthenia gravis. The approval was based on the results of the Phase III…
14 touchdowns. 11.6 yards per carry (on average). 1st Team All-Conference. These accolades show how hard Luke Winterbottom, an all-league running back for Plymouth Whitemarsh High School, worked during…
The Orphan Drug Act of 1983 was implemented after the U.S. Food and Drug Administration observed drug development issues in the rare disease space. Rare diseases often have smaller…
On June 21, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…
When it comes to treating interstitial cystitis, doctors may take a multipronged approach: physical therapy, bladder distention, surgery, nerve stimulation, oral medications. But people with this condition lack therapies…
The U.S. Food and Drug Administration offers various designations to drugs and drug developers to both incentivize medical advancements in the rare disease space and get treatments in the…
In 2015, rare disease stakeholders in North Carolina established the first Rare Disease Advisory Council (RDAC); the National Organization for Rare Disorders (NORD) explains that an RDAC is an…
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