Scientists discover rare genetic condition that attacks kids’ immune systems
The Six Symptoms And Warning Signs Of A Stroke
There are two main types of stroke: ischaemic, which are caused by a blockage in an artery, and haemorrhagic, which is when a blood vessel in the brain ruptures - iStockphoto
Every day in the UK around 240 people suffer the catastrophic impact of a stroke. Sometimes seen as "brain attacks" these happen when blood flow to part of the brain is interrupted, cutting off the oxygen and nutrients our brain cells need to survive.
As brain cells (neurones) can die within minutes, strokes are right up there with heart attacks for being the medical emergencies we most dread.
They can cause profound life-altering disabilities, such as losing the ability to speak or use certain parts of your body. And sadly, one in four of us will suffer one at some point in our lives.
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How to think FAST and recognise the signs of a strokeThere are two main types, ischaemic strokes (accounting for around 87 per cent of all cases) are caused by a blockage (eg a blood clot) in an artery that supplies blood to the brain.
Haemorrhagic strokes (account for 13 per cent) are when a blood vessel in the brain ruptures, causing bleeding and damage to the surrounding brain tissue. The signs of both are the same.
"Early recognition of a stroke is crucial – it can save lives and prevent long-term disability," says Dr Branimir Nevajda, a consultant in neurology and stroke medicine at Basildon University Hospital. He explains how the FAST acronym serves as a quick guide to identifying the signs of a stroke and taking action. "Seek immediate medical attention if you notice any symptoms of a stroke, even if they seem to come and go or they disappear completely," do the following:
FaceAsk the person to smile, can they manage to? Does their face droop on one side?
ArmsAsk them to raise both arms. Check if one arm drifts downwards? Or cannot be raised? You can also check legs.
SpeechAsk them to repeat a simple phrase. Do they sound slurred, garbled or incoherent speech, or find speaking difficult?
TimeAct swiftly – if you see any of these signs call emergency services immediately.
This FAST method has remained unchanged for years, though in some regions around the world, this has been expanded to BE-FAST – which includes additional early warning signs related to balance issues and eye problems:
BalanceCheck for sudden loss of balance or coordination.
EyesCheck for blurred or loss of vision in one or both eyes.
"During a stroke, the brain is deprived of oxygen, and neurones die at a staggering rate – approximately 1.9 million neurones are lost every minute," says Dr Nevajda. "This is why acting quickly is so important in stroke treatment. People need to recognise the signs and get help faster. This expansion of the acronym helps identify strokes that may not show the classic "FAST" signs, and thereby increase the chances of getting timely medical intervention."
SymptomsDr Nevajda explains that each stroke symptom, listed below, gives a clue about what's happening in the brain:
1. Sudden numbness or weaknessThis occurs when a stroke disrupts blood flow to areas of the brain that control muscle movement or sensation. As a result, the affected area of the brain can no longer send or receive proper signals to the corresponding body parts.
2. Trouble speaking or understanding speechStrokes that affect the brain's language centres, such as Broca's area (responsible for speech production) or Wernicke's area (responsible for speech comprehension) disrupt communication pathways, leading to difficulties in speaking clearly or understanding others. This can result in slurred speech or inability to find the right words.
3. Vision problemsIf a stroke occurs anywhere along the visual pathway (i.E. The occipital lobe, the optic nerve, optic pathways or the eye itself) it can cause blurred or double vision or even partial or complete loss of vision in one or both eyes.
4. Severe headacheThis is commonly associated with haemorrhagic strokes. It occurs because bleeding in the brain increases the pressure inside the skull.
5. Dizziness or loss of balanceWhen a stroke affects the posterior regions of the brain (such as the cerebellum or brainstem) that control balance and coordination, it can lead to symptoms of dizziness and vertigo
6. Difficulty walkingStrokes can impair movement in legs, either through weakness or paralysis in the leg muscles themselves (due to damage in the motor cortex) or due to balance problems stemming from affected cerebellar function.
What causes strokes?The underlying cause depends on the type of stroke it is, says Dr Nevajda, but they generally relate to problems affecting the blood circulation or blood vessels. Key causes include:
Causes of Ischaemic strokeAtherosclerosis – the narrowing of arteries due to cholesterol build-up into plaques.
Blood clots – often originating from the heart and especially common in individuals with an irregular heart rhythm called atrial fibrillation.
High blood pressure – longstanding hypertension can lead to the weakening of blood vessel walls, making them more susceptible to rupture.
Aneurysms – these are weak or thin areas in the walls of the blood vessel that can bulge and rupture.
"Up to 80 to 90 per cent of all strokes are preventable and evidence shows that acting to lower blood pressure can significantly reduce the risk it poses to health," says Maeva May, an associate director at the Stroke Association.
High blood pressure contributes to around half of all strokes, making it the single biggest risk factor. Hypertension also affects the heart and causes atrial fibrillation (AF or AFib). This can lead to blood clots forming in the heart, which can often cause strokes. The best way to check for atrial fibrillation is to monitor your pulse; it's normal for occasional beats to be irregular, but if it is continually inconsistent then let your GP know.
Check your blood pressure regularly with a monitor at home, at a pharmacy or at your doctor's surgery. If you are consistently getting readings over 140/90, it is important to let your GP know. For most people, simple lifestyle changes, and sometimes medications, are enough to lower the reading.
Who is most at risk of a stroke?The following factors strongly affect your chance of having a stroke:
Age: Strokes are more frequently seen in individuals over 55.
Gender: Men are more likely to have a stroke than women.
Race: People of Afro-Caribbean descent are at an increased risk.
Medical conditions: Chronic conditions such as high blood pressure, diabetes, high cholesterol, and atrial fibrillation elevate all increase the risk of strokes.
Lifestyle factors: Smoking, excessive alcohol consumption, obesity, diabetes and lack of physical activity all contribute to the risk.
Treatment for strokes depends on the type of stroke and how quickly the patient receives medical attention. Here are the primary treatment approaches:
Ischaemic stroke:Clot-busting medications (thrombolytics): These drugs can dissolve blood clots and restore blood flow. They are only effective when given within a few hours of symptom onset, usually up to 4.5 hours.
Mechanical thrombectomy: This procedure involves using a catheter to physically remove the clot, depending on the patient's condition and the location of the clot itself. It's worth mentioning that this pioneering procedure is recognised as one of the most beneficial procedures in medicine currently.
Surgery: In cases of significant bleeding, surgery may be required to repair the damaged blood vessels or remove excess blood from the brain to relieve pressure within the skull.
Endovascular procedures: These minimally invasive techniques can be used to seal off the bleeding vessel or to repair an aneurysm to reduce the risk of future aneurysmal ruptures.
Rehabilitation: After the initial treatment, many stroke survivors require rehabilitation to recover lost functions. This may involve physical therapy, occupational therapy, and speech therapy to help regain strength, mobility, coordination, and communication skills.
Lifestyle changes: Long-term treatment often involves lifestyle modifications to reduce the risk of future strokes. This involves adopting a healthier diet, increasing physical activity, managing stress, and adhering to prescribed medications for conditions like diabetes, atrial fibrillation, high blood pressure and cholesterol.
Strokes often strike suddenly, but sometimes there are warning signs. The BE-FAST acronym can help recognise early signs of stroke. In addition to these signs, sometimes individuals may experience mini-strokes also known as transient ischaemic attacks (TIAs) before a major stroke. These episodes are temporary, causing stroke-like symptoms that appear suddenly and resolve within a few minutes to a few hours. TIAs serve as critical warning signs, indicating that a full stroke may be imminent (especially in those with pre-existing risk factors). Seeking immediate medical attention can help reduce the risk of a major stroke.
What does a mini-stroke look like?A Transient Ischaemic Attack (TIA) presents with similar symptoms to a full stroke. The key difference is that these symptoms are temporary and completely resolve within minutes to a few hours. More importantly, TIAs do not cause permanent brain damage; however, they are strong indicators of an increased risk for future strokes and provide a window of opportunity for medical interventions.
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The expertsThe Stroke Association supports stroke survivors and their families by phone, at home, and in the community. Find out more at www.Stroke.Org.Uk
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Biotech Stocks Facing FDA Decision In November 2024
(RTTNews) - As October draws to a close, it's time to take a look at the regulatory news that made headlines in the month and look ahead to what's in store for November.
Pfizer's Hympavzi received FDA approval on October 11, for the treatment of Hemophilia, which becomes the first of its kind to work by targeting a protein in the blood clotting process. Astellas followed suit on October 18, with Vyloy, the first CLDN18.2-targeting drug approved in the U.S. For specific gastric cancers. CLDN18.2 is a transmembrane protein. Iterum's Orlynvah received approval on October 25, marking a milestone as the first oral penem approved for use in the U.S. And the second FDA-approved treatment for uncomplicated urinary tract infections in the past two decades.
The FDA has approved 38 novel drugs so far this year (Jan - Oct) compared to 46 during the same time frame last year.
Now let's take a look at the drugs that are at the FDA altar, awaiting a decision in November.
Journey Medical Corp. (DERM)
The FDA decision on Journey Medical's DFD-29, proposed for the treatment of inflammatory lesions and erythema of rosacea in adults, is due on November 4, 2024.
In clinical settings, DFD-29 demonstrated statistical superiority over both the current standard-of-care treatment, Oracea 40 mg capsules, and placebo for Investigator's Global Assessment treatment success as well as the reduction in the total inflammatory lesion count.
If approved, DFD-29 would be the only oral, systemic therapy to address both inflammatory lesions and erythema (redness) from rosacea. The company expects DFD-29 to achieve peak annual net sales exceeding $100 million.
Journey Medical is a partner company of Fortress Biotech Inc. (FBIO).
DERM closed Tuesday's (Oct.29, 2024) trading at $5.74, down 4.33%.
Merus N.V. (MRUS)
Merus' lead drug candidate, Zenocutuzumab (Zeno), proposed for the treatment of patients with neuregulin 1 fusion (NRG1+) non-small cell lung and NRG1+ pancreatic cancer, is under priority review by the FDA.
NRG1 fusions are gene fusions regarded as oncogenic drivers and can be found in various tumor types.
The Biologics License Application for Zeno was accepted by the U.S. Regulatory agency on May 6, 2024. Although a specific decision date hasn't been given, the application is under priority review, which usually means a decision will be made within six months. Therefore, we can expect the decision to be announced by November 6, 2024.
Zenocutuzumab has the potential to be the first and only targeted therapy for patients with NRG1+ lung and pancreatic cancer and may offer a substantial improvement over currently available therapies, according to the company.
MRUS closed Tuesday's trading at $51.92, down 1.70%.
Astellas Pharma Inc. (ALPMY.OB)
Astellas Pharma's supplemental New Drug Application for label update, seeking to include positive 2-year data for its marketed drug IZERVAY for the treatment of geographic atrophy secondary to age-related macular degeneration, awaits the FDA decision on November 19, 2024.
The sNDA was based on results from GATHER2, a phase III clinical trial that evaluated the efficacy and safety of monthly (EM) and every other month (EOM) dosing of IZERVAY through year 2. The GATHER2 data demonstrated that IZERVAY continued to reduce the rate of geographic atrophy lesion growth in patients with geographic atrophy secondary to age-related macular degeneration through 2 years versus sham.
IZERVAY was approved by the FDA on August 4, 2023. During the quarter from April 1, 2024, to June 30, 2024, the drug's sales totaled 12.7 billion yen.
ALPMY.OB closed Tuesday's trading at $11.64, up 3.39%.
PTC Therapeutics Inc. (PTCT)
PTC Therapeutics' Upstaza, a gene therapy for the treatment of AADC deficiency, is under FDA review, with a decision anticipated on November 13, 2024.
Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder that affects the brain. It is characterized by poor muscle tone (hypotonia), delayed development in head control, crawling, speech, Involuntary eye movements, abnormally excessive sweating (hyperhidrosis), Hypersalivation, Drooping eyelids (ptosis), gastrointestinal problems such as reflux, diarrhea, or constipation, behavioral problems, and sleep disturbances, among others.
Upstaza is a one-time gene replacement therapy that is proposed for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype. In clinical trials, Upstaza demonstrated transformational neurological improvements.
This gene therapy, directly administered into the brain, was authorized in the EU to treat adults and children aged 18 months and older with severe AADC deficiency with a genetically confirmed diagnosis, in July 2022, and reportedly carries a list price of about $3.7 million per dose.
PTCT closed Tuesday's trading at $42.50, up 4.99%.
Autolus Therapeutics plc (AUTL)
The FDA decision on Autolus Therapeutics' Obe-cel, proposed for the treatment of patients with relapsed/refractory adult B-cell acute lymphoblastic leukemia, is expected on November 16, 2024.
Obe-cel, also known as Obecabtagene autoleucel, is a CD19 CAR T cell investigational therapy designed to overcome the limitations in clinical activity and safety compared to current CD19 CAR T cell therapies like Bristol Myers' Breyanzi, Gilead's Tecartus and Yescarta, and Novartis' Kymriah.
In a pivotal study, the results of which formed the basis of the Biologics License Application, 78% of patients treated with Obe-cel achieved a complete response (CR) or CR with incomplete hematological recovery (CRi). Analysis of longer follow-up data from the pivotal study, announced in June 2024, showed that 40% of patients were in ongoing remission without subsequent stem cell transplant (SCT) or other intervention.
If approved, Obe-cel will have to compete with Amgen's Blincyto, which generated sales of $508 million in the first half of 2024, an increase of 27% over the year-ago period.
AUTL closed Tuesday's trading at $4.34, down 4.62%.
Applied Therapeutics Inc. (APLT)
Applied Therapeutics' Govorestat, proposed for the treatment of Classic Galactosemia, awaits the FDA decision on November 28, 2024.
Govorestat is a novel Aldose Reductase Inhibitor and a potent and selective compound, which crosses the blood-brain barrier into the Central Nervous System. Aldose Reductase plays a crucial role in the development of Galactosemia.
Galactosemia is a rare, genetic disorder where the body is unable to break down the sugar galactose. Instead, the galactose is converted into galactitol by the enzyme Aldose Reductase, leading to a buildup of this toxic metabolite in tissues and organs resulting in long-term disease complications. There is no known cure or approved treatment for Galactosemia. About 3,000 individuals in the US and 4,000 in Europe are affected by Galactosemia. Classic Galactosemia is the most common subtype of Galactosemia.
Govorestat is also under review by the EMA's Committee for Medicinal Products for Human Use (CHMP). A decision by the EMA is expected early in the first quarter of 2025.
Analysts at Leerink Partners expect Govorestat to achieve global peak sales of roughly $870 million in 2035, assuming it receives approval.
APLT closed Tuesday's trading at $8.75, up 1.39%.
BridgeBio Pharma Inc. (BBIO)
The FDA decision on BridgeBio Pharma's Acoramidis, an investigational drug for the treatment of transthyretin amyloid cardiomyopathy, is due on November 29, 2024.
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare but severe cause of restrictive cardiomyopathy caused by the accumulation of transthyretin fibrils in the myocardium. It can present with new or worsening heart failure or new conduction system disease. (Source: NIH).
In a phase III trial, dubbed ATTRibute-CM, Acoramidis treatment demonstrated an 81% absolute survival rate and a 0.29 observed mean annual cardiovascular-related hospitalization (CVH) frequency, as well as improvements for a large proportion of patients.
If approved, Acoramidis will have to compete with Pfizer's Vyndaqel and Vyndamax, the first treatments for ATTR-CM to receive FDA approval in May 2019. Acoramidis has the potential to hit blockbuster sales at its peak, according to analysts.
BBIO closed Tuesday's trading at $24.50, up 0.25%.
Jazz Pharmaceuticals plc (JAZZ)
The FDA decision on Zanidatamab, the human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody, for the treatment of previously treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer, is due on November 29, 2024.
Zanidatamab is being developed by Jazz Pharmaceuticals and BeiGene, Ltd. (BGNE) under license agreements from Zymeworks Inc. (ZYME), which first developed the molecule.
In the U.S., Europe, and Japan, around 12,000 individuals are diagnosed each year with HER2+ biliary tract cancer (BTC). If approved, Zanidatamab would be the first HER2-targeted therapy specifically for patients with locally advanced or metastatic HER2+ BTC.
According to Jazz Pharma, Zanidatamab could potentially reach peak sales of $2 billion.
JAZZ closed Tuesday's trading at $111.91, up 0.48%.
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