August is SMA Awareness Month!
August has begun and you know what that means – it is Spinal Muscular Atrophy (SMA) Awareness Month! During this month, many patients, advocacy groups, family members, and others work…
Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
August has begun and you know what that means – it is Spinal Muscular Atrophy (SMA) Awareness Month! During this month, many patients, advocacy groups, family members, and others work…
Note: Research Square publishes preliminary, non-peer-reviewed scientific reports. Therefore, while the data below is interesting, it is not academically published and, as stated within the published article on Research Square: "the…
Currently, the standards-of-care for treating medulloblastoma include surgical interventions, chemotherapy, and radiation. Finding new and effective treatment options would help to improve patient outcomes. According to an article in The…
According to a news release from biotechnology company Sound Pharmaceuticals, enrollment has begun in the Phase 3 STOPMD-3 clinical trial. So far, the first patient has been enrolled – with…
In recent years, there have been advances in the treatment of pancreatic cancer. This is crucial, as the prognosis for this cancer is often poor, and the cancer itself is…
In the United States, Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions. A rare condition is one affecting fewer…
Currently, it is possible to screen for Down syndrome during pregnancy. However, the screening measure used – ultrasound imagery – has less than 80% detection accuracy using common indicators,…
In 2019, Rian and Mike Fee celebrated the birth of their son Anderson, who they call Andy. Since birth, Andy has always been such a light in the lives…
1On July 27, 2022, global biopharmaceutical company GSK plc shared some exciting news via news release. The company’s therapy Benlysta (belimumab), designed for pediatric patients with lupus nephritis, is now…
2157 words (source) vs. 423 words (mine) - 5% match In the Phase 2 LILAC clinical trial, researchers sought to evaluate the safety, efficacy, and tolerability of litifilimab (BIIB059) for…
At the end of July 2022, biotechnology company Biogen Inc. shared via news release some exciting updates in regards to its therapy tofersen, an investigational treatment for patients with…
When Ryker Colón was first born, his parents Eddie and Brandi noticed something potentially concerning. Their young son had a chronic cough - and nobody could seem to figure out…
Those with multiple myeloma within the European Union will soon have access to a new therapeutic option. An article for Cancer Network shares that the European Commission (EC) approved…
Oftentimes, data and research play crucial roles in understanding more about both a disease or illness, and also how it impacts those affected. This is especially important in the rare…
Jason and Rachel Moore were always excited to start a family. Currently, the pair has two children – Naomi, age 21 months, and Lacey, age 4 months. But their journey…
This year, the European Hematology Association (EHA) held its Hybrid Conference starting on June 15; if registered for the conference, you may access the Congress Platform until August 15, 2022.…
Will teclistamab soon be available for patients with multiple myeloma within the European Union? According to OncLive, approval seems to be on the horizon. Following results from the Phase ½…
The HealthWell Foundation is a nonprofit organization with a mission to reduce financial barriers to care for underinsured patients with chronic or life-altering diseases. The foundation assists with copays,…
Following the approval of carfilzomib (Kyprolis) for relapsed or refractory (R/R) multiple myeloma (MM) in Japan around two years ago, researchers sought to understand the safety and efficacy of the…
Myalgic encephalomyelitis, or chronic fatigue syndrome (ME/CFS), can cause sustained, extreme, and often debilitating fatigue that does not improve with rest. Currently, no approved therapies exist for those with ME/CFS.…
In late June 2022, Patient Worthy discussed how doctors and scientists were concerned about rising rates of nonalcoholic fatty liver disease (NAFLD) in Filipinos. But according to new research, the…
When Stormi Vanden Bosch and Tyler Scotting found out they were expecting another son, they were thrilled. On October 31st, 2019, they welcomed their son Baylor to the world.…
For months, those within the rare disease community have been watching to see whether Upstaza (eladocagene exuparvovec) would be approved for use within the European Union. In fact, in…
A Phase 2 clinical trial which sought to evaluate the safety, efficacy, and tolerability of NP-120 (ifenprodil) for idiopathic pulmonary fibrosis (IPF) had some surprising results. Initially, the study…
Clinical trials are an important tool in medical research to understand more about the safety, efficacy, and tolerability of therapeutics - and how these may influence patient outcomes. Recently,…