Bone marrow failure

• “The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder.
• Currently, there are no existing options to potentially prevent BMF for patients with FA,” said Kinnari Patel, Pharm.D., MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma.
• The safety profile was highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated.
• The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024.

• It is the largest and most visible organ in the human body.
• Being the most visible of our organs, the skin also offers us a view into the body tissues that it protects.
• So don’t think of your skin merely aesthetically – think of it as a reflection of your health.

Bullseye

• But while the vast majority of tick bites won’t make you ill, there is one rash that should prompt a visit to your doctor if you spot it.
• Erythema migrans, a rash named for its ability to rapidly expand across the skin, is a hallmark of Lyme disease, a potentially severe bacterial illness.
• This rash forms a classic target pattern, like a bullseye on a dartboard.

Purpura

• Some rashes are given a colourful namesake – purpura is one such example.
• Purpura refers to a rash of small purple or red dots.
• Purpura signals an issue with either the walls of the tiny blood vessels that feed the skin or the blood within them.

Skin spiders

Skin rashes can also take on recognisable shapes. Spider naevi represent an issue within skin arterioles (small arteries which supply the skin with blood). Arterioles open and close to control the loss of heat from the body’s surface. But sometimes they can get stuck open – and a spider-like pattern will appear.

• Crush the body under a fingertip and the whole thing disappears, as your touch temporarily stops the blood flow.
• Treat the underlying cause, and the spiders often vanish with time – though they may persist or reappear later.

Black velvet

• This “black velvet” skin appearance is more commonly seen in darker skins.
• Usually, the condition is associated with disorders of the metabolism – namely type 2 diabetes and polycystic ovary syndrome.

Butterfly rashes

• Cardiac valves have the important role of correctly directing the journey of blood through the heart and preventing backflow.
• The body’s natural response is to preserve core blood volume, shutting off flow towards the skin.
• The net effect can produce a purple-red rash, high across the cheeks and the bridge of the nose, like the outstretched wings of a butterfly.

Dan Baumgardt does not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.

• The data will be featured in a presentation at the 2023 Fanconi Anemia Research Fund (FARF) Scientific Symposium, taking place on September 28 – October 1, 2023, in Vancouver, Canada.
• The ongoing investigator initiated Phase 1/2 clinical trial is evaluating a conditioning regimen that includes briquilimab as a potential treatment for FA patients in bone marrow failure.
• “All three FA patients who underwent conditioning with briquilimab achieved full blood count recovery.
• These outcomes underscore the potential for briquilimab conditioning regimen to redefine the landscape of FA therapy.

• The "Polycythemia Vera - Epidemiology Forecast - 2032" report has been added to ResearchAndMarkets.com's offering.
• The prevalence is projected to increase during the forecasted period
  In the United States, there were approximately 69,000 males and about 38,000 females affected with PV in 2022.
• This 'Polycythemia Vera (PV)- Epidemiology Forecast-2032' report delivers an in-depth understanding of the disease, historical and forecasted epidemiology in the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan.
• Polycythemia Vera is a rare blood disease in which the body makes excessive red blood cells.