Muscular Dystrophy Pakistan

14/04/2026

Limb Girdle Muscular Dystrophy (LGMD)

Limb Girdle Muscular Dystrophy (LGMD) is a group of inherited (genetic) disorders that cause progressive muscle weakness. It mainly affects the muscles around the shoulders (shoulder girdle) and hips (pelvic girdle), which are essential for movement and stability.

Key Symptoms

Weakness in upper arms and thighs

Difficulty walking, climbing stairs, or lifting objects

Frequent falls

Trouble standing up from a sitting position

Gradual muscle wasting (loss of muscle mass)

In some cases, heart and breathing complications

Types

LGMD includes many subtypes based on genetic causes. It is broadly classified into:

Autosomal Dominant (LGMD D): Inherited from one parent

Autosomal Recessive (LGMD R): Inherited when both parents carry the gene

Causes

LGMD is caused by mutations in specific genes responsible for maintaining healthy muscle function. These mutations lead to muscle damage and weakness over time.

Diagnosis

Blood tests (e.g., elevated creatine kinase levels)

Genetic testing

Electromyography (EMG)

Muscle biopsy

Treatment

There is currently no cure for LGMD, but treatments can help manage symptoms and improve quality of life:

Physiotherapy and regular exercise

Assistive devices (braces, wheelchairs)

Cardiac and respiratory monitoring

Supportive care and rehabilitation

Care and Management

Maintain a balanced diet

Engage in safe, guided physical activity

Regular medical check-ups

Prevent infections and complications

11/04/2026

We are pleased to inform you that after dedicated efforts over the past few weeks, we have successfully developed and launched our new professional registration system. This marks an important step forward in strengthening our organizational structure and improving service delivery.

We warmly invite you to visit the platform, complete your registration, and obtain your unique registration ID. This ID will serve as your official identification within our system and will be used for all future records, communications, and access to services and benefits.

All upcoming facilities, support programs, and opportunities will be provided based on the data collected through this system. Therefore, we strongly encourage you to complete your registration at the earliest and become an active part of this initiative.

Registration Link:
https://musculardystrophypakistanform.netlify.app

Thank you for your continued support and cooperation.

Warm regards,
Muscular Dystrophy Pakistan

10/04/2026

On behalf of Muscular Dystrophy Pakistan, I am honored to convey my message on the occasion of the Rare Disease Event held at Aga Khan University Hospital (AKUH), Karachi...

07/04/2026

🇵🇰 Gratitude to Rare Diseases International (RDI)

Muscular Dystrophy Pakistan (MDP) sincerely thanks Rare Diseases International (RDI) for welcoming us as an Associate Member.

This recognition is a meaningful step forward for our mission and a powerful encouragement for our ongoing efforts to support individuals and families affected by muscular dystrophies and rare neuromuscular disorders in Pakistan.

We deeply appreciate this opportunity to be part of a global network committed to advocacy, collaboration, and improving the lives of rare disease communities worldwide. We look forward to contributing actively and working together to amplify patient voices and create lasting impact.

Thank you for your trust and support.

06/04/2026

🇵🇰 A Proud Milestone for Pakistan’s Rare Disease Community

We are honored to share that Muscular Dystrophy Pakistan (MDP) has been welcomed as an Associate Member of RDI (Rare Diseases International) — marking a significant step forward for rare disease advocacy in Pakistan.

MDP remains committed to improving the lives of individuals and families affected by muscular dystrophies and rare neuromuscular disorders across the country. Through impactful awareness campaigns, advocacy initiatives, patient support services, research collaboration, and capacity building, we strive to promote early diagnosis, enhance access to quality healthcare, and advocate for inclusive health policies.

This membership strengthens our connection with global networks, enabling us to amplify patient voices, share knowledge, and bring international attention to the needs of Pakistan’s rare disease community.

Together, we are building a more inclusive, informed, and hopeful future.

04/04/2026

Update for LGMD 2i Community of Pakistan
By Muscular Dystrophy Pakistan

We are pleased to share an important and hopeful update for the LGMD 2i (LGMDR9) community.

BridgeBio Pharma has submitted an application to the U.S. FDA for approval of BBP-418, a potential oral treatment. If approved, it could become the first-ever therapy for LGMD 2i and a major breakthrough for all LGMD types.

Key Highlights: • Improves muscle function by targeting glycosylation
• Clinical trials show better walking ability and lung function
• Granted Fast Track & Orphan Drug status
• Expected approval: Late 2026 / Early 2027

What it means for Pakistan:
This development brings new hope for patients and families, showing strong global progress toward treatment access.

Our Commitment:
Muscular Dystrophy Pakistan remains dedicated to awareness, advocacy, and support for all MD patients.

Together, we stay hopeful and strong.

Muscular Dystrophy Pakistan

30/03/2026

We are pleased to inform you that we have successfully completed the development of our Information, Education, and Communication (IEC) materials, including brochures, flyers, booklets, and banners. These resources have been designed to support effective community engagement, awareness, and data collection.

We are now fully prepared to initiate project activities. Insha Allah, we will officially begin implementation from 1st April.

Thank you EveryLife Foundation for Rare Diseases

30/03/2026

🌐 Turning Challenges into Opportunities: Advancing Global Collaboration in Rare Diseases

Rare Diseases International (RDI) participated in the 6ᵗʰ International Conference on Rare Diseases, organized by its member organization, RARE DISEASES GREECE. The conference brought together leading international and national experts, researchers, global leaders from academia, healthcare professionals, policy-makers, industry leaders, and patient representatives, all united by a shared mission: “Building a brighter future together — transforming the challenges of rare diseases into national and international opportunities.” This year’s event provided an inclusive platform for dialogue and action across Europe and beyond, exploring how cooperation among governments, scientific communities, and patient organizations can accelerate access to diagnosis, treatment, and sustainable care for people living with rare conditions.

Monica McClain, Global Programmes Senior Manager at Rare Diseases International, participated in Session 1: “Build for Impact: Landmark Partnerships for Rare Diseases.” The session focused on ERDERA and HLM-RARE initiatives, Integration of European Reference Networks (ERNs) into national systems, including lessons from JARDIN, Cross-ERN collaboration in areas such as data, diagnostics, ePAGs, and education. The panel was moderated by Ana Rath (Commissioner RDI-LCRD) and featured distinguished speakers including Daria Julkowska, Maurizio Scarpa, Holm Graessner (Commissioner RDI-LCRD), Ines Hernando, and Monica McClain.

The discussion emphasized the importance of strong partnerships, integrated systems, and cross-border collaboration to drive meaningful impact for the rare disease community. Key takeaways included:

🔬 ERNs, ERDERA and EURORDIS-Rare Diseases Europe collaborations are improving the access to care and treatment and standardizing data collection.

🧑‍🧑‍🧒‍🧒 Patient involvement and empowerment are key drivers of sustainable progress via the development of the Global Action Plan on Rare Diseases (GAPRD).

28/03/2026

25/03/2026

To learn more about LGMD, please visit our website: http://LGMD-Info.org

21/03/2026

May the light of Eid illuminate your path and bring peace to your home. Sending prayers of health and joy to every MD patient and caregiver.

Eid Mubarak from:

Muscular Dystrophy Pakistan.