Biogen’s Post

Sometimes I have the privilege to work in disease areas people have rarely heard of, for conditions I hope no one ever has to have. Progressive Familial Intrahepatic Cholestasis (PFIC) is an umbrella term that represents a group of rare genetic liver disorders. The hallmark symptom of PFIC is a severe and debilitating itch. The disease typically presents in infancy or early childhood. The exact prevalence is unknown, but PFIC is estimated to affect 1 in 50,000-100,000 people worldwide. There is currently no cure for PFIC and related diseases. Learn more by visiting www.pfic.org. Together, we can make a difference. #StrongerTogether #ItchingForACure

29 February, which only occurs every 4 years, is Rare Disease Day. A disease is considered as rare if no more than 5 in 10’000 people suffer from it. Around 8’000 rare diseases are known to date and more are discovered every year.    Healthcare Communication is all about profound information for doctors and patients. Puls also creates communication materials for various rare diseases. These materials can help to raise awareness for these diseases.     Do you know PNH? Probably not. PNH stands for paroxysmal nocturnal haemoglobinuria and describes an acquired genetic disease that is very rare. There are around 16 cases per million inhabitants. It mainly manifests itself as anaemia and an increased tendency to thrombosis and should always be taken seriously. #puls_healthcommunication #pharmamarketing #healthcarecommunication #healthcaremarketing #basel #rarediseaseday #raredisease FEEL OUR PULSE? Website | puls.ch Instagram | puls_ag X | puls_ag

QurAlis presents! Excited to have our CSO Dan Elbaum as a featured speaker at the 2nd Annual Neurodegeneration Targets conference taking place next week. Dan's talk is entitled, "Recent Advances in the Development of Splice-Switching #Oligonucleotides for #CNS Diseases." #precisionmedicine #neurodegenerativediseases

29 February, which only occurs every 4 years, is Rare Disease Day. A disease is considered as rare if no more than 5 in 10’000 people suffer from it. Around 8’000 rare diseases are known to date and more are discovered every year.    Healthcare Communication is all about profound information for doctors and patients. Puls also creates communication materials for various rare diseases. These materials can help to raise awareness for these diseases.     Do you know PNH? Probably not. PNH stands for paroxysmal nocturnal haemoglobinuria and describes an acquired genetic disease that is very rare. There are around 16 cases per million inhabitants. It mainly manifests itself as anaemia and an increased tendency to thrombosis and should always be taken seriously. #puls_healthcommunication #pharmamarketing #healthcarecommunication #healthcaremarketing #basel #rarediseaseday #raredisease FEEL OUR PULSE? Website | puls.ch Instagram | puls_ag X | puls_ag

Rare Disease Day is tomorrow, Thursday, February 29. Its purpose is to raise awareness for rare diseases, and improve access to treatment and medical representation for individuals with rare diseases and their families. At SequenceMD, our primary focus is on the testing, diagnosis and management of unresolved symptoms, undiagnosed conditions and rare diseases for patients of all ages. If you, a family member or friend have had health issues that continue without answers — or have been diagnosed with a rare disease — now’s the time to get to the root of what’s going on with your health. A genetic diagnosis can reveal insights into your medical history, prepare you for what’s ahead, and inform a customized, precision care plan to maintain or improve your health.  LEARN MORE HERE: https://lnkd.in/ePGHRdnd #rarediseaseday #genetictesting #genetics #sequencemd #caremanagement #diagnosis #raredisorders #neurogenetic #raregeneticdisorders #metabolicconditions #geneticdisorders

Today, IMol joins the global community in recognizing Rare Disease Day. We appreciate the importance of awareness about the challenges faced by individuals with rare diseases. Our research aims to develop treatments for mitochondrial diseases.   #RareDiseaseDay #IMol #ResearchForRareDiseases Polska Akademia Nauk NCN Narodowe Centrum Nauki Ministerstwo Nauki i Szkolnictwa Wyższego

29 February, which only occurs every 4 years, is Rare Disease Day. A disease is considered as rare if no more than 5 in 10’000 people suffer from it. Around 8’000 rare diseases are known to date and more are discovered every year.    Healthcare Communication is all about profound information for doctors and patients. Puls also creates communication materials for various rare diseases. These materials can help to raise awareness for these diseases.     Do you know PNH? Probably not. PNH stands for paroxysmal nocturnal haemoglobinuria and describes an acquired genetic disease that is very rare. There are around 16 cases per million inhabitants. It mainly manifests itself as anaemia and an increased tendency to thrombosis and should always be taken seriously. #puls_healthcommunication #pharmamarketing #healthcarecommunication #healthcaremarketing #basel #rarediseaseday #raredisease FEEL OUR PULSE? Website | puls.ch Instagram | puls_ag X | puls_ag

A rare disease is a disease that affects a small percentage of the population. Most rare diseases are genetic in origin hence they are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases usually appear early in life, and about 30% of children with rare diseases will die before the age of 5. Some of the most common rare diseases include: Ehlers –Danlos Syndrome (EDS), Sickle Cell, Cystic Fibrosis, Duchenne Muscular Dystrophy (DMD) & Haemophilia. Sources: https://lnkd.in/g4ZZ4SS5 https://lnkd.in/gpH56N4R #rare_diseases #pharmacy #fip #fipecpg #ipsf Want to explore the ECPG's #opportunities & #events for #Members? Check here: https://lnkd.in/dS6YMfU2

29 February, which only occurs every 4 years, is Rare Disease Day. A disease is considered as rare if no more than 5 in 10’000 people suffer from it. Around 8’000 rare diseases are known to date and more are discovered every year.    Healthcare Communication is all about profound information for doctors and patients. Puls also creates communication materials for various rare diseases. These materials can help to raise awareness for these diseases.     Do you know PNH? Probably not. PNH stands for paroxysmal nocturnal haemoglobinuria and describes an acquired genetic disease that is very rare. There are around 16 cases per million inhabitants. It mainly manifests itself as anaemia and an increased tendency to thrombosis and should always be taken seriously. #puls_healthcommunication #pharmamarketing #healthcarecommunication #healthcaremarketing #basel #rarediseaseday #raredisease FEEL OUR PULSE? Website | puls.ch Instagram | puls_ag X | puls_ag

Rare diseases are conditions that afflict less than 200,000 people in the United States, but they are far less rare in the aggregate, afflicting millions—and in their disparate types: There are more than 10,000 known rare diseases. In his latest essay in The San Diego Union-Tribune, David Brenner discusses the particular challenges of identifying and developing treatments for rare diseases, and the work being done at the Sanford Children’s Health Research Center. Read more: https://bit.ly/48ccbQz Mark your calendars: February 29 is Rare Disease Day. Sanford Burnham Prebys will host its annual scientific and family symposium on congenital disorders of glycosylation, an umbrella term that covers more than 190 rare (fewer than 2,000 children worldwide) genetic diseases on March 1, 2 and 3. Register here: https://bit.ly/494vPPu #rarediease #rarediseaseawareness #rarediseaseresearch #rarediseaseday #childrenshealth