Updated Study Data Suggests Efruxifermin Benefits Stage 2 or 3 MASH
source: shutterstock.com

Updated Study Data Suggests Efruxifermin Benefits Stage 2 or 3 MASH

Over the past few years, Akero Therapeutics has experienced high highs and low lows in its efforts to advance efruxifermin, an investigational drug for people living with metabolic dysfunction-associated steatohepatitis…

Continue Reading Updated Study Data Suggests Efruxifermin Benefits Stage 2 or 3 MASH
Highlights of ACMG 2024
source: shutterstock.com

Highlights of ACMG 2024

Perhaps 70- 85% of rare diseases have a root in genetics, - which is why continuous research and reporting is so critical for those with rare conditions. This is exactly…

Continue Reading Highlights of ACMG 2024
The Power of Positive Thoughts on Our Health
source: shutterstock.com

The Power of Positive Thoughts on Our Health

Most people have probably heard the saying, “change your thoughts, change your life.” There is great truth to this because how we think can significantly impact our mental and physical…

Continue Reading The Power of Positive Thoughts on Our Health
Rare Community Profiles: A Mother-Son Pair Discuss the Importance of Wellbeing in Managing NF2-Related Schwannomatosis (NF2)
Jo and Oscar. Photo courtesy of Jo Ward

Rare Community Profiles: A Mother-Son Pair Discuss the Importance of Wellbeing in Managing NF2-Related Schwannomatosis (NF2)

  Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

Continue Reading Rare Community Profiles: A Mother-Son Pair Discuss the Importance of Wellbeing in Managing NF2-Related Schwannomatosis (NF2)
Study Lists Neurological Disease as the Top Cause of Disability and Illness Around the World
source: shutterstock.com

Study Lists Neurological Disease as the Top Cause of Disability and Illness Around the World

Neurological conditions now have the highest rates of disability and ill health worldwide. The Disparity Although more than 80% of the neurological deaths and nervous system disorders occur in lower…

Continue Reading Study Lists Neurological Disease as the Top Cause of Disability and Illness Around the World
Rare Community Profiles: Yellow for Yiannis: Angela’s Mission to Advance Research into IRF2BPL
Fotocitizen / Pixabay

Rare Community Profiles: Yellow for Yiannis: Angela’s Mission to Advance Research into IRF2BPL

  Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

Continue Reading Rare Community Profiles: Yellow for Yiannis: Angela’s Mission to Advance Research into IRF2BPL
Rare Community Profiles: A Mother’s Journey to Raise 4H Leukodystrophy POLR3B Awareness and Change Her Son’s Life
source: pixabay.com

Rare Community Profiles: A Mother’s Journey to Raise 4H Leukodystrophy POLR3B Awareness and Change Her Son’s Life

  Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

Continue Reading Rare Community Profiles: A Mother’s Journey to Raise 4H Leukodystrophy POLR3B Awareness and Change Her Son’s Life
ICYMI: AIT-101 Earned Orphan Drug Designation in the EU for ALS in March 2024
Capri23auto / Pixabay

ICYMI: AIT-101 Earned Orphan Drug Designation in the EU for ALS in March 2024

Historically, it has been difficult to incentivize companies to develop therapies for rare or “orphan” conditions. The National Conference of State Legislatures (NCSL) explains that “orphan” conditions are: neglected conditions…

Continue Reading ICYMI: AIT-101 Earned Orphan Drug Designation in the EU for ALS in March 2024
Phase 2 Study Evaluates Nizubaglustat for Niemann-Pick Disease type C
Pexels / Pixabay

Phase 2 Study Evaluates Nizubaglustat for Niemann-Pick Disease type C

There are limited therapeutic interventions for those living with Niemann-Pick disease type C (NPC), a rare and progressive genetic disorder. Unfortunately, many people with NPC experience severe and life-threatening complications…

Continue Reading Phase 2 Study Evaluates Nizubaglustat for Niemann-Pick Disease type C
Rare Community Profiles: Could It Be HCM? A Mother-Daughter Duo Raise Hypertrophic Cardiomyopathy Awareness
klimkin / Pixabay

Rare Community Profiles: Could It Be HCM? A Mother-Daughter Duo Raise Hypertrophic Cardiomyopathy Awareness

  Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

Continue Reading Rare Community Profiles: Could It Be HCM? A Mother-Daughter Duo Raise Hypertrophic Cardiomyopathy Awareness
Achieving Cushing’s Disease Remission After Surgery Could Increase Risk of New-Onset Autoimmune Disease
source: pixabay.com

Achieving Cushing’s Disease Remission After Surgery Could Increase Risk of New-Onset Autoimmune Disease

People with Cushing's disease have a benign pituitary tumor or pituitary growth that, when triggered, overproduced adrenocorticotropic hormone (ACTH). Too much ACTH causes the body to also overproduce cortisol. Known as…

Continue Reading Achieving Cushing’s Disease Remission After Surgery Could Increase Risk of New-Onset Autoimmune Disease