Latest news with #SMA
Deccan Herald
3 days ago
- Health
- Deccan Herald
Spinal Muscular Atrophy: Debilitating and costly
Fifteen years ago, Rashmi Sampath noticed that her seven-month-old daughter Deeksha couldn't independently sit and stand. After multiple specialist visits, tests and treatment options explored over one and a half years, Deeksha was diagnosed with Spinal Muscular Atrophy (SMA), a progressive neurodegenerative disease leading to gradual muscle wasting. 'We did a DNA test, and that is how we learnt about SMA. Had we known to recognise signs earlier, maybe the treatment could have been started earlier,' said Rashmi. In 2021, Deeksha, on a wheelchair since she was eight years old, started taking Risdiplam, an orally administered drug for SMA, which costs about Rs 6.2 lakh per bottle. Had her medication costs not been covered under a compassionate access program by Roche Pharma, it would have been difficult for her family to afford her treatment. .Generic drugs can bring hope for SMA patients'.Keerthana, another child with SMA, also had treatment delays, impacting her progress. Her parents, H Nagashree and Kishore, both government employees, have to spend Rs 50-75 lakh annually for the treatment of the two-year-old. 'We are getting three bottles free for the cost of two, but it is still quite expensive for us. She needs 4.4 mg of the oral solution every day. We need two bottles of 60 mg a month, since one bottle lasts about 18 days. We buy two at once and pay about Rs 12.4 lakh and get three more free with them', said Kishore. 'After a year, only two bottles will be given for free. These costs will increase when Keerthana's weight increases and the dosage will go up,' said Nagashree, who lamented that they had not yet received any government support so far. .The Delhi High Court's March 2025 order allowing Hyderabad-based Natco Pharma to develop a generic version of Risdiplam and rejecting the plea of patent holder Swiss pharma major F Hoffmann-La Roche Limited for an injunction has brought hope for over two lakh SMA patients. Roche has now appealed to the Supreme Court. If Natco is successful, it can offer Risdiplam for an estimated cost of Rs 15,900 per 60 mg bottle However, doctors, families of patients and activists argued that this might not be enough. They urged the government to step in and talk with the manufacturer and bulk buy the medicine to reduce the annual costs of the families of SMA patients. .'If a family has a strong background of SMA cases, we suggest parents undergo genetic testing and counselling to rule out the possibility of SMA. Even if nobody in the family has disease manifestations at all, but we suspect SMA in the baby, we do nerve conduction studies and electroneuromyography (ENMG) tests,' Dr Shiva Kumar HR, a consultant neurologist in Bengaluru, said. Dr Kumar noted that deaths occur due to complications arising out of the disorder, not the disorder itself. 'Over time, it weakens respiratory muscles, causing patients to die due to Type II respiratory failure. Life expectancy can be prolonged with gene therapy, but it cannot be called a cure because once a degeneration has begun, it cannot be reversed,' he said. .Prasanna Kumar Shirol, founder of the Organisation for Rare Diseases India (ORDI), said that although the government had the National Policy for Rare Diseases, 2021, its assistance was limited to the one-time payment of Rs 50 lakh per patient. 'Sustainable funding is the only way out. With an ICMR-rated 90% treatment effectiveness for SMA, government support, regardless of the cost, is essential,' he said, adding that not only the Centre but the state governments too needed to step in. 'The governments can develop a standard operating procedure for every rare condition like the SMA, identifying what is the inclusion and exclusion criteria for treatment, the criteria for extending treatment. They can then get into talks with the pharma companies, figure out drug supply for a fixed period and monitor drug effectiveness with timely reviews,' suggested Shirol.
Deccan Herald
3 days ago
- Health
- Deccan Herald
Generic drugs can bring hope for SMA patients'
Dr Ann Agnes Mathew, a consultant paediatric neurologist and neuromuscular specialist based in Bengaluru, treats nearly 600 SMA patients from across India. In an interview with DH's Udbhavi Balakrishna, she highlighted the challenges in diagnosing SMA and shared her views on the expensive treatment of the disease.
Irish Independent
4 days ago
- Politics
- Irish Independent
Historic Mayo mansion will no longer accommodate Ukrainian refugees
Ballinafad House, which accommodated over 30 beneficiaries of Temporary Protection (at one point, will revert to alternative use next month. The 110-room property, owned by Australian Bede Tannock, has accommodated Ukrainian refugees since 2022. The historic property had been providing accommodation and operated as a venue for weddings, civil ceremonies and public gatherings. The Department of Justice has confirmed that the property, and Hotel Newport Holiday Apartments, while revert to original or alternative use. Eight people are affected by the discontinuation of the use of Ballinafad House while eleven were living in the Hotel Newport Holiday Apartments. Approximately 2,400 Ukrainian refugees across the country are to be affected by the change of purpose of 30 properties. The Department of Justice said the contracts with private accommodation providers were not being renewed partly due to a 'reduced need' for such facilities to accommodate BOTPs. 'Many people are choosing to move on from State-supported accommodation or are leaving Ireland,' the department stated. 'State accommodation contracts may also be ending where compliance issues arise, or where the owner chooses to end their contract.' Those residing in Ballinafad House must relocate by June 6 while those in the Hotel Newport Holiday Apartments must relocate by July 4. It is understood that many of those who had been living in Ballinafad House have now left the property. Built near the village of Belcarra in 1827, Ballinafad House had fallen into disrepair when restoration works began there in March 2014. ADVERTISEMENT The 70,000 square foot property featured on RTE'S 'Great House Revival' and boasts 40 bedrooms, its own chapel and handball court. The five-bay two-storey-over-raised-basement mansion was built by Maurice Blake, a former High Sheriff of County Mayo, who hailed from a well-known Catholic family who pioneered Gaelic games in the locality. In 1908, Maurice and Anne Blake's son, Lieutenant Colonel Count Llewellyn Joseph, donated the building to the Society of African Missions (SMA). The SMA used the building as a secondary school and minor seminary, known as The Sacred Heart College, to prepare students for missionary priesthood as more ancillary buildings were built onto the house. Over 500 priests were ordained there before the seminary closed in 1957. The property was later purchased by Balla Mart, who used it used as an agricultural college. It closed due to a lack of funding in the mid-1970s after five years in operation. This forced 80 boarders and 120 day students to continue their education elsewhere. Plans for a 'detention centre' and a 'multi-million-euro sports complex' at Ballinafad had been reported in The Mayo News years before Mr Tannock acquired the property for €80,000 in 2014. In 2019, Ballinafad House was placed on the market for €2 million.
New Straits Times
4 days ago
- Health
- New Straits Times
Sustainable path for rare disease medicine access
ACCESS to innovative medicines remains a challenge in Malaysia's public healthcare sector. As of March 2023, the Health Ministry identified nearly 500 rare diseases, yet many patients still struggle to receive proper treatment. A study by the School of Pharmaceutical Sciences at Universiti Sains Malaysia and Universiti Malaya Medical Centre found that only 60 per cent of rare disease patients in Malaysia receive treatment. This alarming statistic highlights the urgent need for a unified approach, involving public-private collaboration and the adoption of progressive access models to ensure affordability and long-term sustainability. It is equally important for the ministry to explore new treatment access strategies to guarantee equitable healthcare for all, including those living with rare diseases. Building on Progress in Medicine Access The ministry has made strides in improving medicine access, particularly with the introduction of the Medicines Access Scheme (MASc) guideline. This framework provides a cleargovernance structure for the application, evaluation, and approval of Patient Assistance Schemes (PASc), effectively addressing longstanding administrative challenges. In the past, PASc's implementation was hindered by concerns over the ethical implications of free-of-charge (FOC) stock provisions tied to medicine purchases. With these regulatory uncertainties now resolved, patients can benefit from a more structured and reliable pathway to access essential treatments, reducing reliance on out-of-pocket expenses or external funding sources. Malaysia has made steady progress in advancing treatment options for rare diseases, including the approval of several disease-modifying therapies (DMTs) for spinal muscular atrophy (SMA) in recent years. Building on this foundation, the country recently reached a major milestone with the approval of its first gene therapy for SMA, signalling Malaysia's entry into the era of high-value, transformative medicines. Gene therapy presents a new frontier for SMA management, offering the potential for improved prognosis and enhanced quality of life. Clinical studies for SMA DMT have demonstrated unprecedented improvements in survival, reduced dependence on nutritional and respiratory support, and the achievement and maintenance of key motor milestones such as sitting, standing, and walking - outcomes rarely seen in the natural progression of the disease. These findings underscore the broad impact of gene therapy beyond symptomatic relief, offering meaningful functional gains for individuals living with SMA. However, long-term accessibility requires more than just expanding healthcare coverage. To fully realise the benefits of these groundbreaking treatments, Malaysia must establish sustainable access models to ensure that patients can accessthese life-changing treatments without barriers. Sustainable Solution: Innovative Access Models Globally, healthcare systems are adopting progressive access frameworks to address the rising cost of innovative treatments. Outcome-based and pay-for-performance models have gained traction for high-cost conditions, as they align payment with real-world effectiveness and protect public budgets from unwarranted expenditures. For example, Argentina established a dedicated reimbursement fund for SMA in 2023, managed through the National Commission for Evaluation of Health Technologies. This centralised, expert-led mechanism assesses treatment eligibility on a case-by-case basis, promoting governance, transparency, and equitable distribution. A similar fund in Malaysia - anchored in expert oversight and transparent eligibility criteria - could support equitable access to treatment for rare disease patients. In Australia, a unique payer licence agreement was developed to enable universal access to hepatitis C treatment while capping the government's annual reimbursement liability. This budget-certain model offers a relevant blueprint for Malaysia in managing newer, high-cost rare disease therapies. Applying such models to orphan diseases could create opportunities to scale access without exceeding fiscal constraints. Malaysia has also seen success through the Malaysia Patient Assistance Program (MyPAP), which enabled universal access to medicines for chronic myeloid leukaemia (CML) patients via cost-sharing among the government and pharmaceutical partners. Building on this model, supported by philanthropic or CSR contributions, could unlock access for conditions where treatment affordability remains a barrier. Countries such as Taiwan and Brazil have further advanced outcome-based reimbursement by tying treatment payment to long-term clinical milestones. In Taiwan, SMA gene therapy is reimbursed via a structured agreement that incorporates expert panel reviews, milestone assessments, and extended monitoring over a decade. Brazil's system similarly connects staged payments to patient progress. These frameworks not only safeguard public investment but also prioritise treatment effectiveness, offering valuable direction for the ministry's Pharmaceutical Services Programme (PSP) and public payers seeking to manage high-value therapy access responsibly. Public-Private Collaboration: The Way Forward As advanced therapies become more widely available, financial aid programs alone are insufficient. Malaysia must establish a comprehensive framework that ties funding to patient outcomes, integrating structured reimbursement models, outcome-based agreements, and risk-sharing frameworks. Strong public-private collaboration will be crucial to implementing these strategies effectively, with each stakeholder playing a vital role in expanding access. While the government ensures regulatory oversight and sustainable reimbursement structures, private stakeholders contribute to financing and delivering these therapies, creating a more balanced and efficient system. Although the ministry has made progress in improving governance through the MASc guideline, deeper engagement with private stakeholders - including pharmaceutical companies, insurers and non-governmental organisations - is critical for developing tailored financing mechanisms. Building on this progress, the MASc can be expanded to include flexible access schemes, such as outcome-based models, which link reimbursement to patient health outcomes. This approach would align payment structures with treatment effectiveness, ensuring patients receive the most appropriate therapies. Strengthening these collaborations will empower Malaysia to implement sustainable access solutions that improve treatment affordability, optimize healthcare spending, and position the country as a regional hub for investment in life sciences. Seizing the Opportunity for Change The RM25 million allocation for rare disease treatment in Budget 2025 presents a crucial opportunity to pilot new financing structures. By adopting a proven access model and fostering transparent collaboration across sectors, Malaysia can take meaningful steps toward bridging the treatment gap for rare disease patients, ensuring timely access to life-saving therapies. Without a proactive approach, thousands of patients risk being left without the life-saving treatments they desperately need. Ensuring access to high-value medicines is not just a healthcare challenge, but a moral imperative. The time for action is now. Malaysia must seize this moment to build a more inclusive, patient-centred healthcare system, especiallyfor the benefit of those suffering from rare diseases. *The writer is the managing director of Datametrics Research and Information Centre Sdn Bhd.
National Post
4 days ago
- Business
- National Post
SmartStop Receives BBB Credit Rating From Morningstar DBRS
Article content LADERA RANCH, Calif. — SmartStop Self Storage REIT, Inc. ('SmartStop') (NYSE: SMA), an internally managed real estate investment trust and a premier owner and operator of self-storage facilities in the United States and Canada, announced that DBRS, Inc. ('Morningstar DBRS') has assigned an Issuer Rating of BBB with a Stable trend to SmartStop OP, L.P., the operating partnership of SmartStop. The rating was based on the credit risk profile of SmartStop and its subsidiaries (including SmartStop OP, L.P.). Morningstar DBRS also assigned a senior unsecured debt rating on SmartStop's 2032 Private Placement Notes of BBB with a stable trend. Article content Article content The rating is supported by information submitted to Morningstar DBRS as of May 21, 2025, including management presentations, financial forecasts, annual reports, and organizational documentation. Article content Assumptions underlying the rating include the expectation that any future senior unsecured debt issued by SmartStop OP, L.P. will be unsubordinated and rank equally with its other unsecured obligations. These obligations are also expected to be fully guaranteed by SmartStop and its subsidiaries, consistent with the guarantees provided under its existing credit agreement and in accordance with Morningstar DBRS' Global Corporate Criteria. Article content About SmartStop Self Storage REIT, Inc. (SmartStop): Article content SmartStop Self Storage REIT, Inc. ('SmartStop') (NYSE:SMA) is a self-managed REIT with a fully integrated operations team of approximately 590 self-storage professionals focused on growing the SmartStop® Self Storage brand. SmartStop, through its indirect subsidiary SmartStop REIT Advisors, LLC, also sponsors other self-storage programs. As of May 28, 2025, SmartStop has an owned or managed portfolio of 220 operating properties in 23 states, the District of Columbia, and Canada, comprising approximately 157,100 units and 17.7 million rentable square feet. SmartStop and its affiliates own or manage 41 operating self-storage properties in Canada, which total approximately 34,400 units and 3.5 million rentable square feet. Additional information regarding SmartStop is available at Article content Article content Article content Article content Article content
